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Posts Tagged ‘myocardial infarction’

Arteriogenesis and Cardiac Repair: Two Biomaterials – Injectable Thymosin beta4 and Myocardial Matrix Hydrogel

Curator: Aviva Lev-Ari, PhD, RN

 

Thymosin beta 4 (Tβ4)

is a highly conserved, 43-amino acid acidic peptide (pI 4.6) that was first isolated from bovine thymus tissue over 25 years ago. It is present in most tissues and cell lines and is found in high concentrations in blood platelets, neutrophils, macrophages, and other lymphoid tissues. Tβ4 has numerous physiological functions, the most prominent of which being the regulation of actin polymerization in mammalian nucleated cells and with subsequent effects on actin cytoskeletal organization, necessary for cell motility, organogenesis, and other important cellular events.

Recently,

  • Tβ4 was shown to be expressed in the developing heart and found to stimulate migration of cardiomyocytes and endothelial cells, promote survival of cardiomyocytes (Nature, 2004), and most recently
  • to play an essential role in all key stages of cardiac vessel development: vasculogenesis, angiogenesis, and arteriogenesis (Nature 2006).

These results suggest that Tβ4 may have significant therapeutic potential in humans to protect myocardium and promote cardiomyocyte survival in the acute stages of ischemic heart disease.

RegeneRx Biopharmaceuticals, Inc. is developing Tβ4 for the treatment of patients with acute myocardial infarction (AMI). Such efforts presented will include the formulation, development, and manufacture of a suitable drug product for use in the clinic, the performance of nonclinical pharmacology and toxicology studies, and the implementation of a phase 1 clinical protocol to assess the safety, tolerability, and the pharmacokinetics of Tβ4 in healthy volunteers.

 

SOURCE:
EXPLORATIONS with THYMOSIN beta4 FOR INDUCING ADULT EPICARDIAL PROGENETOR MOBILIZATION AND NEOVASCULARIZATION is presented in
Resident-cell-based Therapy in Human Ischaemic Heart Disease: Evolution in the PROMISE of Thymosin beta4 for Cardiac Repair

http://pharmaceuticalintelligence.com/2012/04/30/93/

Clinical Study Data of Thymosin beta 4 Presented

Published on October 3, 2009 at 5:10 AM

REGENERX BIOPHARMACEUTICALS, INC. (NYSE Amex:RGN) today reported on several clinical studies with Thymosin beta 4 (Tβ4) presented the Second International Symposium on Thymosins in Health and Disease, in Catania, Italy. The following are synopses of the presentations:

Myocardial Development of RGN-352 (Injectable Tβ4 Peptide)

David Crockford, RegeneRx’s vice president for clinical and regulatory affairs presented an overview of the biological properties that support Tβ4’s near term and long term clinical applications. Mr. Crockford noted that special emphasis is being placed on the development of RGN-352 for the systemic (injectable) treatment of patients with ST-elevation myocardial infarction (STEMI) in combination with percutaneous coronary intervention, the current standard of care in most western countries for this common type of heart attack. The goal with RGN-352 is to prevent or repair continued damage to cardiac tissue post-heart attack, when such tissue around the damaged site remains at risk.

Dr. Dennis Ruff, vice president and medical director of ICON, and principal investigator, presented the most current results on the Phase I safety study with RGN-352 entitled, “A Randomized, Double-blind, Placebo-controlled, Dose-response Phase I Study of the Safety and Tolerability of the Intravenous Administration of Thymosin Beta 4 and its Pharmacokinetics After Single and Multiple Doses in Healthy Volunteers.” Dr. Ruff discussed key aspects of the study and concluded with, “There were no dose limiting or serious adverse events throughout the dosing period. Synthetic Tβ4 administered intravenously up to 1260 mg, and for up to 14 days, appears to be well tolerated with low incidence of adverse events and no evidence of serious adverse events.”

http://www.news-medical.net/news/20091003/Clinical-study-data-of-Thymosin-beta-4-presented.aspx

RegeneRx Receives Notice of Allowance from Chinese Patent Office for Treatment and Prevention of Heart Disease

RegeneRx Receives Notice of Allowance from Chinese Patent Office for Treatment and Prevention of Heart Disease

February 7, 2013 — Rockville, Md.

RegeneRx Biopharmaceuticals, Inc. (OTC Bulletin Board: RGRX) (“the Company” or “RegeneRx”) today announced that it has received a Notice of Allowance of a Chinese patent application for uses of Thymosin beta 4 (TB4) for treating, preventing, inhibiting or reducing heart tissue deterioration, injury or damage in a subject with heart failure disease. Claims also include uses for restoring heart tissue in those subjects. The patent will expire July 26, 2026.

http://www.regenerx.com/wt/page/pr_1360265259

Active Research on Thymosins in Cardiovascular Disease Reported in 2010 and 2012 Annual Conference on Thymosins, Proceedings by NY Academy of Sciences

Use of the cardioprotectants thymosin β4 and dexrazoxane during congenital heart surgery: proposal for a randomized, double-blind, clinical trial

Neonates and infants undergoing heart surgery with cardioplegic arrest experience both inflammation and myocardial ischemia-reperfusion (IR) injury. These processes provoke myocardial apoptosis and oxygen-free radical formation that result in cardiac injury and dysfunction. Thymosin β4 (Tβ4) is a naturally occurring peptide that has cardioprotective and antiapoptotic effects. Similarly, dexrazoxane provides cardioprotection by reduction of toxic reactive oxygen species (ROS) and suppression of apoptosis. We propose a pilot pharmacokinetic/safety trial of Tβ4 and dexrazoxane in children less than one year of age, followed by a randomized, double-blind, clinical trial of Tβ4 or dexrazoxane versus placebo during congenital heart surgery. We will evaluate postoperative time to resolution of organ failure, development of low cardiac output syndrome, length of cardiac ICU and hospital stays, and echocardiographic indices of cardiac dysfunction. Results could establish the clinical utility of Tβ4 and/or dexrazoxane in ameliorating ischemia-reperfusion injury during congenital heart surgery.[1]

Cardiac repair with thymosin β4 and cardiac reprogramming factors

Heart disease is a leading cause of death in newborns and in adults. We previously reported that the G-actin–sequestering peptide thymosin β4 promotes myocardial survival in hypoxia and promotes neoangiogenesis, resulting in cardiac repair after injury. More recently, we showed that reprogramming of cardiac fibroblasts to cardiomyocyte-like cells in vivo after coronary artery ligation using three cardiac transcription factors (Gata4/Mef2c/Tbx5) offers an alternative approach to regenerate heart muscle. We have combined the delivery of thymosin β4 and the cardiac reprogramming factors to further enhance the degree of cardiac repair and improvement in cardiac function after myocardial infarction. These findings suggest that thymosin β4 and cardiac reprogramming technology may synergistically limit damage to the heart and promote cardiac regeneration through the stimulation of endogenous cells within the heart.[2]

NMR structural studies of thymosin α1 and β-thymosins

Thymosin proteins, originally isolated from fractionation of thymus tissue, represent a class of compounds that we now know are present in numerous other tissues, are unrelated to each other in a genetic sense, and appear to have different functions within the cell. Thymosin α1 (generic drug name thymalfasin; trade name Zadaxin) is derived from a precursor molecule, prothymosin, by proteolytic cleavage, and stimulates the immune system. Although the peptide is natively unstructured in aqueous solution, the helical structure has been observed in the presence of trifluoroethanol or unilamellar vesicles, and these studies are consistent with the presence of a dynamic helical structure whose sides are not completely hydrophilic or hydrophobic. This helical structure may occur in circulation when the peptide comes into contact with membranes. In this report, we discuss the current knowledge of the thymosin α1 structure and similar properties of thymosin β4 and thymosin β9, in different environments.[3]

Thymosin β4 sustained release from poly (lactide-co-glycolide) microspheres: synthesis and implications for treatment of myocardial ischemia

 A sustained release formulation for the therapeutic peptide thymosin β4 (Tβ4) that can be localized to the heart and reduce the concentration and frequency of dose is being explored as a means to improve its delivery in humans. This review contains concepts involved in the delivery of peptides to the heart and the synthesis of polymer microspheres for the sustained release of peptides, including Tβ4. Initial results of poly(lactic-co-glycolic acid) microspheres synthesized with specific tolerances for intramyocardial injection that demonstrate the encapsulation and release of Tβ4 from double-emulsion microspheres are also presented.[4]
Thymosin β4 and cardiac repair
Hypoxic heart disease is a predominant cause of disability and death worldwide. As adult mammals are incapable of cardiac repair after infarction, the discovery of effective methods to achieve myocardial and vascular regeneration is crucial. Efforts to use stem cells to repopulate damaged tissue are currently limited by technical considerations and restricted cell potential. We discovered that the small, secreted peptide thymosin β4 (Tβ4) could be sufficiently used to inhibit myocardial cell death, stimulate vessel growth, and activate endogenous cardiac progenitors by reminding the adult heart on its embryonic program in vivo. The initiation of epicardial thickening accompanied by increase of myocardial and epicardial progenitors with or without infarction indicate that the reactivation process is independent of injury. Our results demonstrate Tβ4 to be the first known molecule able to initiate simultaneous myocardial and vascular regeneration after systemic administration in vivo. Given our findings, the utility of Tβ4 to heal cardiac injury may hold promise and warrant further investigation.[7]
Thymosin β4 facilitates epicardial neovascularization of the injured adult heart
Ischemic heart disease complicated by coronary artery occlusion causes myocardial infarction (MI), which is the major cause of morbidity and mortality in humans (http://www.who.int/cardiovascular_diseases/resources/atlas/en/index.html). After MI the human heart has an impaired capacity to regenerate and, despite the high prevalence of cardiovascular disease worldwide, there is currently only limited insight into how to stimulate repair of the injured adult heart from its component parts. Efficient cardiac regeneration requires the replacement of lost cardiomyocytes, formation of new coronary blood vessels, and appropriate modulation of inflammation to prevent maladaptive remodeling, fibrosis/scarring, and consequent cardiac dysfunction. Here we show that thymosin β4 (Tβ4) promotes new vasculature in both the intact and injured mammalian heart. We demonstrate that limited EPDC-derived endothelial-restricted neovascularization constitutes suboptimal “endogenous repair,” following injury, which is significantly augmented by Tβ4 to increase and stabilize the vascular plexus via collateral vessel growth. As such, we identify Tβ4 as a facilitator of cardiac neovascularization and highlight adult EPDCs as resident progenitors which, when instructed by Tβ4, have the capacity to sustain the myocardium after ischemic damage.[8]
Thymosin β4 enhances repair by organizing connective tissue and preventing the appearance of myofibroblasts
Incisional wounds in rats treated locally with thymosin β4 (Tβ4) healed with minimal scaring and without loss in wound breaking strength. Treated wounds were significantly narrower in width. Polarized light microscopy treated wounds had superior organized collagen fibers, displaying a red birefringence, which is consistent with mature connective tissue. Control incisions had randomly organized collagen fibers, displaying green birefringence that is consistent with immature connective tissue. Immunohistology treated wounds had few myofibroblasts and fibroblasts with α smooth muscle actin (SMA) stained stress fibers. Polyvinyl alcohol sponge implants placed in subcutaneous pockets received either carrier or 100 μg of Tβ4 on days 2, 3, and 4. On day 14, treated implants revealed longer, thicker collagen fiber bundles with intense yellow-red birefringence by polarized light microscopy. In controls, fine, thin collagen fiber bundles were arranged in random arrays with predominantly green birefringence. Controls contained mostly myofibroblasts, while few myofibroblasts appeared in Tβ4 treated implants. Electron microscopy confirmed both cell types and the degree of collagen fiber bundle organization. Our results demonstrate that Tβ4 treated wounds appear to mature earlier and heal with minimal scaring.[9]
Thymosin β4: a key factor for protective effects of eEPCs in acute and chronic ischemia
Acute myocardial infarction is still one of the leading causes of death in the industrial nations. Even after successful revascularization, myocardial ischemia results in a loss of cardiomyocytes and scar formation. Embryonic EPCs (eEPCs), retroinfused into the ischemic region of the pig heart, provided rapid paracrine benefit to acute and chronic ischemia in a PI-3K/Akt-dependent manner. In a model of acute myocardial ischemia, infarct size and loss of regional myocardial function decreased after eEPC application, unless cell pre-treatment with thymosin β4 shRNA was performed. Thymosin ß4 peptide retroinfusion mimicked the eEPC-derived improvement of infarct size and myocardial function. In chronic ischemia (rabbit model), eEPCs retroinfused into the ischemic hindlimb enhanced capillary density, collateral growth, and perfusion. Therapeutic neovascularization was absent when thymosin ß4 shRNA was introduced into eEPCs before application. In conclusion, eEPCs are capable of acute and chronic ischemia protection in a thymosin ß4 dependent manner. [10]
Thymosin β4: a candidate for treatment of stroke?
Neurorestorative therapy is the next frontier in the treatment of stroke. An expanding body of evidence supports the theory that after stroke, certain cellular changes occur that resemble early stages of development. Increased expression of developmental proteins in the area bordering the infarct suggest an active repair or reconditioning response to ischemic injury. Neurorestorative therapy targets parenchymal cells (neurons, oligodendrocytes, astrocyes, and endothelial cells) to enhance endogenous neurogenesis, angiogenesis, axonal sprouting, and synaptogenesis to promote functional recovery. Pharmacological treatments include statins, phosphodiesterase 5 inhibitors, erythropoietin, and nitric oxide donors that have all improved funtional outcome after stroke in the preclinial arena. Thymosin β4 (Tβ4) is expressed in both the developing and adult brain and it has been shown to stimulate vasculogenesis, angiogenesis, and arteriogenesis in the postnatal and adult murine cardiac myocardium. In this manuscript, we describe our rationale and techniques to test our hypothesis that Tβ4 may be a candidate neurorestorative agent. [11]
Prothymosin α as robustness molecule against ischemic stress to brain and retina

Following stroke or traumatic damage, neuronal death via both necrosis and apoptosis causes loss of functions, including memory, sensory perception, and motor skills. As necrosis has the nature to expand, while apoptosis stops the cell death cascade in the brain, necrosis is considered to be a promising target for rapid treatment for stroke. We identified the nuclear protein, prothymosin alpha (ProTα) from the conditioned medium of serum-free culture of cortical neurons as a key protein-inhibiting necrosis. In the culture of cortical neurons in the serum-free condition without any supplements, ProTα inhibited the necrosis, but caused apoptosis. In the ischemic brain or retina, ProTα showed a potent inhibition of both necrosis and apoptosis. By use of anti-brain-derived neurotrophic factor or anti-erythropoietin IgG, we found that ProTα inhibits necrosis, but causes apoptosis, which is in turn inhibited by ProTα-induced neurotrophins under the condition of ischemia. From the experiment using anti-ProTα IgG or antisense oligonucleotide for ProTα, it was revealed that ProTα has a pathophysiological role in protecting neurons in stroke.[12]

 
Thymosin β4 and cardiac repair
Hypoxic heart disease is a predominant cause of disability and death worldwide. As adult mammals are incapable of cardiac repair after infarction, the discovery of effective methods to achieve myocardial and vascular regeneration is crucial. Efforts to use stem cells to repopulate damaged tissue are currently limited by technical considerations and restricted cell potential. We discovered that the small, secreted peptide thymosin β4 (Tβ4) could be sufficiently used to inhibit myocardial cell death, stimulate vessel growth, and activate endogenous cardiac progenitors by reminding the adult heart on its embryonic program in vivo. The initiation of epicardial thickening accompanied by increase of myocardial and epicardial progenitors with or without infarction indicate that the reactivation process is independent of injury. Our results demonstrate Tβ4 to be the first known molecule able to initiate simultaneous myocardial and vascular regeneration after systemic administration in vivo. Given our findings, the utility of Tβ4 to heal cardiac injury may hold promise and warrant further investigation.[13]
Thymosin β4 facilitates epicardial neovascularization of the injured adult heart
schemic heart disease complicated by coronary artery occlusion causes myocardial infarction (MI), which is the major cause of morbidity and mortality in humans (http://www.who.int/cardiovascular_diseases/resources/atlas/en/index.html). After MI the human heart has an impaired capacity to regenerate and, despite the high prevalence of cardiovascular disease worldwide, there is currently only limited insight into how to stimulate repair of the injured adult heart from its component parts. Efficient cardiac regeneration requires the replacement of lost cardiomyocytes, formation of new coronary blood vessels, and appropriate modulation of inflammation to prevent maladaptive remodeling, fibrosis/scarring, and consequent cardiac dysfunction. Here we show that thymosin β4 (Tβ4) promotes new vasculature in both the intact and injured mammalian heart. We demonstrate that limited EPDC-derived endothelial-restricted neovascularization constitutes suboptimal “endogenous repair,” following injury, which is significantly augmented by Tβ4 to increase and stabilize the vascular plexus via collateral vessel growth. As such, we identify Tβ4 as a facilitator of cardiac neovascularization and highlight adult EPDCs as resident progenitors which, when instructed by Tβ4, have the capacity to sustain the myocardium after ischemic damage. [14]
Thymosin β4: a key factor for protective effects of eEPCs in acute and chronic ischemia

Acute myocardial infarction is still one of the leading causes of death in the industrial nations. Even after successful revascularization, myocardial ischemia results in a loss of cardiomyocytes and scar formation. Embryonic EPCs (eEPCs), retroinfused into the ischemic region of the pig heart, provided rapid paracrine benefit to acute and chronic ischemia in a PI-3K/Akt-dependent manner. In a model of acute myocardial ischemia, infarct size and loss of regional myocardial function decreased after eEPC application, unless cell pre-treatment with thymosin β4 shRNA was performed. Thymosin ß4 peptide retroinfusion mimicked the eEPC-derived improvement of infarct size and myocardial function. In chronic ischemia (rabbit model), eEPCs retroinfused into the ischemic hindlimb enhanced capillary density, collateral growth, and perfusion. Therapeutic neovascularization was absent when thymosin ß4 shRNA was introduced into eEPCs before application. In conclusion, eEPCs are capable of acute and chronic ischemia protection in a thymosin ß4 dependent manner.[15]

 
Thymosin β4: a candidate for treatment of stroke?
Neurorestorative therapy is the next frontier in the treatment of stroke. An expanding body of evidence supports the theory that after stroke, certain cellular changes occur that resemble early stages of development. Increased expression of developmental proteins in the area bordering the infarct suggest an active repair or reconditioning response to ischemic injury. Neurorestorative therapy targets parenchymal cells (neurons, oligodendrocytes, astrocyes, and endothelial cells) to enhance endogenous neurogenesis, angiogenesis, axonal sprouting, and synaptogenesis to promote functional recovery. Pharmacological treatments include statins, phosphodiesterase 5 inhibitors, erythropoietin, and nitric oxide donors that have all improved funtional outcome after stroke in the preclinial arena. Thymosin β4 (Tβ4) is expressed in both the developing and adult brain and it has been shown to stimulate vasculogenesis, angiogenesis, and arteriogenesis in the postnatal and adult murine cardiac myocardium. In this manuscript, we describe our rationale and techniques to test our hypothesis that Tβ4 may be a candidate neurorestorative agent.[16]
Thymosin β4: structure, function, and biological properties supporting current and future clinical applications

Published studies have described a number of physiological properties and cellular functions of thymosin β4 (Tβ4), the major G-actin-sequestering molecule in mammalian cells. Those activities include the promotion of cell migration, blood vessel formation, cell survival, stem cell differentiation, the modulation of cytokines, chemokines, and specific proteases, the upregulation of matrix molecules and gene expression, and the downregulation of a major nuclear transcription factor. Such properties have provided the scientific rationale for a number of ongoing and planned dermal, corneal, cardiac clinical trials evaluating the tissue protective, regenerative and repair potential of Tβ4, and direction for future clinical applications in the treatment of diseases of the central nervous system, lung inflammatory disease, and sepsis. A special emphasis is placed on the development of Tβ4 in the treatment of patients with ST elevation myocardial infarction in combination with percutaneous coronary intervention.[17]

The effect of thymosin treatment of venous ulcers

Venous ulcers are responsible for about 70% of the chronic ulcers of the lower limbs. Standard of care includes compression, dressings, debridement of devitalized tissue, and infection control. Thymosin beta 4 (Tβ4), a synthetic copy of the naturally occurring 43 amino-acid peptide, has been found to have wound healing and anti-inflammatory properties, and is thought to exert its therapeutic effect through promotion of keratinocyte and endothelial cell migration, increased collagen deposition, and stimulation of angiogenesis. To assess the safety, tolerability, and efficacy of topically administered Tβ4 in patients with venous stasis ulcers, a double-blind, placebo-controlled, dose-escalation study was conducted in eight European sites (five in Italy and three in Poland) that enrolled and randomized 73 patients. The safety profile of all doses of administered Tβ4 was deemed acceptable and comparable to placebo. Efficacy findings from this Phase 2 study suggest that a Tβ4 dose of 0.03% may have the potential to accelerate wound healing and that complete wound healing can be achieved within 3 months in about 25% of the patients, especially among those whose wounds are small to moderate in size or mild to moderate in severity.[18]

A randomized, placebo-controlled, single and multiple dose study of intravenous thymosin β4 in healthy volunteers

Synthetic thymosin beta 4 (Tβ4) may have a potential use in promoting myocardial cell survival during acute myocardial infarction. Four cohorts, with 10 healthy subjects each, were given a single intravenous dose of placebo or synthetic Tβ4. Cohorts received ascending doses of either 42, 140, 420, or 1260 mg. Following safety review, subjects were given the same dose regimen daily for 14 days. Safety evaluations, incidence of Treatment-Emergent Adverse Events, and pharmacokinetic parameters were evaluated. Adverse events were infrequent, and mild or moderate in intensity. There were no dose limiting toxicities or serious adverse events. Pharmacokinetic profile for single dose showed a dose proportional response, and an increasing half-life with increasing dose. Synthetic Tβ4 given intravenously as a single dose or in multiple daily doses for 14 days over a dose range of 42–1260 mg was well tolerated with no evidence of dose limiting toxicity. Further development for use in cardiac ischemia should be considered.[19]

Safety and Efficacy of an Injectable Extracellular Matrix Hydrogel for Treating Myocardial Infarction

  1. Sonya B. Seif-Naraghi1,*,
  2. Jennifer M. Singelyn1,*,
  3. Michael A. Salvatore2,
  4. Kent G. Osborn1,
  5. Jean J. Wang1,
  6. Unatti Sampat1,
  7. Oi Ling Kwan1,
  8. G. Monet Strachan1,
  9. Jonathan Wong3,
  10. Pamela J. Schup-Magoffin1,
  11. Rebecca L. Braden1,
  12. Kendra Bartels1,
  13. Jessica A. DeQuach2,
  14. Mark Preul4,
  15. Adam M. Kinsey2,
  16. Anthony N. DeMaria1,
  17. Nabil Dib1 and
  18. Karen L. Christman1,

+Author Affiliations

  1. 1University of California, San Diego, La Jolla, CA 92093, USA.
  2. 2Ventrix, Inc., San Diego, CA 92109, USA.
  3. 3Biologics Delivery Systems, Irwindale, CA 91706, USA.
  4. 4Barrow Neurological Institute, Phoenix, AZ 85013, USA.

+Author Notes

  • * These authors contributed equally to this work.
  1. †To whom correspondence should be addressed. E-mail: christman@eng.ucsd.edu

ABSTRACT

New therapies are needed to prevent heart failure after myocardial infarction (MI). As experimental treatment strategies for MI approach translation, safety and efficacy must be established in relevant animal models that mimic the clinical situation. We have developed an injectable hydrogel derived from porcine myocardial extracellular matrix as a scaffold for cardiac repair after MI. We establish the safety and efficacy of this injectable biomaterial in large- and small-animal studies that simulate the clinical setting. Infarcted pigs were treated with percutaneous transendocardial injections of the myocardial matrix hydrogel 2 weeks after MI and evaluated after 3 months. Echocardiography indicated improvement in cardiac function, ventricular volumes, and global wall motion scores. Furthermore, a significantly larger zone of cardiac muscle was found at the endocardium in matrix-injected pigs compared to controls. In rats, we establish the safety of this biomaterial and explore the host response via direct injection into the left ventricular lumen and in an inflammation study, both of which support the biocompatibility of this material. Hemocompatibility studies with human blood indicate that exposure to the material at relevant concentrations does not affect clotting times or platelet activation. This work therefore provides a strong platform to move forward in clinical studies with this cardiac-specific biomaterial that can be delivered by catheter.

  • Copyright © 2013, American Association for the Advancement of Science
Citation: S. B. Seif-Naraghi, J. M. Singelyn, M. A. Salvatore, K. G. Osborn, J. J. Wang, U. Sampat, O. L. Kwan, G. M. Strachan, J. Wong, P. J. Schup-Magoffin, R. L. Braden, K. Bartels, J. A. DeQuach, M. Preul, A. M. Kinsey, A. N. DeMaria, N. Dib, K. L. Christman, Safety and Efficacy of an Injectable Extracellular Matrix Hydrogel for Treating Myocardial Infarction.

RELATED RESOURCES ON SCIENCE SITES

In Science Translational Medicine

REFERENCES OF THYMOSIN IN CARDIOVASCULAR DISEASE

Thymosins in Health and Disease II: 3rd International Symposium on The Emerging Clinical Applications of Tymosin beta 4 in Cardiovascular Disease

Annals of the New York Academy of Sciences, October 2012 Volume 1270 Pages vii-ix, 1–121.

Allan L. Goldstein, Enrico Garaci, Editors, Thymosins in Cardiovascular Disease, November 2012, Wiley-Blackwell

http://onlinelibrary.wiley.com/doi/10.1111/nyas.2012.1270.issue-1/issuetoc

http://www.wiley.com/WileyCDA/WileyTitle/productCd-1573319104.html?cid=RSS_WILEY2_LIFEMED

1


Use of the cardioprotectants thymosin β4 and dexrazoxane during congenital heart surgery: proposal for a randomized, double-blind, clinical trial (pages 59–65) Daniel Stromberg, Tia Raymond, David Samuel, David Crockford, William Stigall, Steven Leonard, Eric Mendeloff and Andrew Gormley
Article first published online: 10 OCT 2012 | DOI: 10.1111/j.1749-6632.2012.06710.x

2


Cardiac repair with thymosin β4 and cardiac reprogramming factors (pages 66–72) Deepak Srivastava, Masaki Ieda, Jidong Fu and Li Qian
Article first published online: 10 OCT 2012 | DOI: 10.1111/j.1749-6632.2012.06696.x

3 NMR structural studies of thymosin α1 and β-thymosins (pages 73–78) David E. Volk, Cynthia W. Tuthill, Miguel-Angel Elizondo-Riojas and David G. Gorenstein
Article first published online: 10 OCT 2012 | DOI: 10.1111/j.1749-6632.2012.06656.x

4

Thymosin β4 sustained release from poly(lactide-co-glycolide) microspheres: synthesis and implications for treatment of myocardial ischemia (pages 112–119) Jeffrey E. Thatcher, Tré Welch, Robert C. Eberhart, Zoltan A. Schelly and J. Michael DiMaio
Article first published online: 10 OCT 2012 | DOI: 10.1111/j.1749-6632.2012.06681.x

5 Corrigendum for Ann. N.Y. Acad. Sci. 2012. 1254: 57–65 (page 121) Article first published online: 10 OCT 2012 | DOI: 10.1111/j.1749-6632.2012.06793.x
This article corrects:
A bird’s-eye view of cell therapy and tissue engineering for cardiac regeneration
Vol. 1254, Issue 1, 57–65, Article first published online: 30 APR 2012

Thymosins in Health and Disease: 2nd International Symposium,
Annals of the New York Academy of Sciences, May 2010 Volume 1194 Pages ix–xi, 1–230 

http://onlinelibrary.wiley.com/doi/10.1111/nyas.2010.1194.issue-1/issuetoc

6. Preface to Thymosins in Health and Disease (pages ix–xi) Enrico Garaci and Allan L. Goldstein
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05493.x

7.
Thymosin β4 and cardiac repair (pages 87–96) Santwana Shrivastava, Deepak Srivastava, Eric N. Olson, J. Michael DiMaio and Ildiko Bock-Marquette
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05468.x

8.
Thymosin β4 facilitates epicardial neovascularization of the injured adult heart (pages 97–104) Nicola Smart, Catherine A. Risebro, James E. Clark, Elisabeth Ehler, Lucile Miquerol, Alex Rossdeutsch, Michael S. Marber and Paul R. Riley
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05478.x

9.
Thymosin β4 enhances repair by organizing connective tissue and preventing the appearance of myofibroblasts (pages 118–124) H. Paul Ehrlich and Sprague W. Hazard III
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05483.x

10. Thymosin β4: a key factor for protective effects of eEPCs in acute and chronic ischemia (pages 105–111) Rabea Hinkel, Ildiko Bock-Marquette, Antonis K. Hazopoulos and Christian Kupatt
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05489.x
Corrected by:
Corrigendum for Ann. N. Y. Acad. Sci. 1194: 105–111
Vol. 1205, Issue 1, 284, Article first published online: 14 SEP 2010

11.

Thymosin β4: a candidate for treatment of stroke? (pages 112–117) Daniel C. Morris, Michael Chopp, Li Zhang and Zheng G. Zhang
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05469.x

12. Prothymosin α as robustness molecule against ischemic stress to brain and retina (pages 20–26) Hiroshi Ueda, Hayato Matsunaga, Hitoshi Uchida and Mutsumi Ueda
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05466.x

13.
Thymosin β4 and cardiac repair (pages 87–96) Santwana Shrivastava, Deepak Srivastava, Eric N. Olson, J. Michael DiMaio and Ildiko Bock-Marquette
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05468.x

14.

Thymosin β4 facilitates epicardial neovascularization of the injured adult heart (pages 97–104) Nicola Smart, Catherine A. Risebro, James E. Clark, Elisabeth Ehler, Lucile Miquerol, Alex Rossdeutsch, Michael S. Marber and Paul R. Riley
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05478.x

15.

Thymosin β4: a key factor for protective effects of eEPCs in acute and chronic ischemia (pages 105–111) Rabea Hinkel, Ildiko Bock-Marquette, Antonis K. Hazopoulos and Christian Kupatt
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05489.x
Corrected by:
Corrigendum for Ann. N. Y. Acad. Sci. 1194: 105–111
Vol. 1205, Issue 1, 284, Article first published online: 14 SEP 2010

16.

Thymosin β4: a candidate for treatment of stroke? (pages 112–117) Daniel C. Morris, Michael Chopp, Li Zhang and Zheng G. Zhang
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05469.x

17.Thymosin β4: structure, function, and biological properties supporting current and future clinical applications (pages 179–189) David Crockford, Nabila Turjman, Christian Allan and Janet Angel
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05492.x

18.

The effect of thymosin treatment of venous ulcers (pages 207–212) G. Guarnera, A. DeRosa and R. Camerini, on behalf of 8 European sites
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05490.x

19.
A randomized, placebo-controlled, single and multiple dose study of intravenous thymosin β4 in healthy volunteers (pages 223–229) Dennis Ruff, David Crockford, Gino Girardi and Yuxin Zhang
Article first published online: 3 MAY 2010 | DOI: 10.1111/j.1749-6632.2010.05474.x

Other related articles on this Open Access Online Scientific Journal include the following:

Gene Therapy Into Healthy Heart Muscle: Reprogramming Scar Tissue In Damaged Hearts

http://pharmaceuticalintelligence.com/2013/01/09/gene-therapy-into-healthy-heart-muscle-reprogramming-scar-tissue-in-damaged-hearts/

Human Embryonic-Derived Cardiac Progenitor Cells for Myocardial Repair

http://pharmaceuticalintelligence.com/2012/08/01/human-embryonic-derived-cardiac-progenitor-cells-for-myocardial-repair/

Human embryonic pluripotent stem cells and healing post-myocardial infarction

http://pharmaceuticalintelligence.com/2012/08/07/human-embryonic-pluripotent-stem-cells-and-healing-post-myocardial-infarction/

Resident-cell-based Therapy in Human Ischaemic Heart Disease: Evolution in the PROMISE of Thymosin beta4 for Cardiac Repair

http://pharmaceuticalintelligence.com/2012/04/30/93/

Heart Renewal by pre-existing Cardiomyocytes: Source of New Heart Cell Growth Discovered

http://pharmaceuticalintelligence.com/2012/12/23/heart-renewal-by-pre-existing-cardiomyocytes-source-of-new-heart-cell-growth-discovered/

Absorb™ Bioresorbable Vascular Scaffold: An International Launch by Abbott Laboratories

http://pharmaceuticalintelligence.com/2012/09/29/absorb-bioresorbable-vascular-scaffold-an-international-launch-by-abbott-laboratories/

Heart patients’ skin cells turned into healthy heart muscle cells

http://pharmaceuticalintelligence.com/2012/06/04/heart-patients-skin-cells-turned-into-healthy-heart-muscle-cells/

Telling NO to Cardiac Risk

http://pharmaceuticalintelligence.com/2012/12/10/telling-no-to-cardiac-risk/

Read Full Post »

Introducing Dr. Tim Wu – Interventional Cardiologist, Inventor and Entrepreneur

 

Author: Ed Kislauskis, PhD

Article ID #18: Introducing Dr. Tim Wu – Interventional Cardiologist, Inventor and Entrepreneur. Published on 1/14/2013

WordCloud Image Produced by Adam Tubman

 

Welcome readers to the first in a series of interviews with future scientific leaders in biotechnology and medicine.  In this post I interview a close colleague and clinical scientist who appears to be on a fast-track to achieving his vision for the future of interventional cardiology – at the very vanguard of applied nanotechnology.

Tim (Tiangen) Wu, M.D has graciously accepted my invitation to answer a few questions about how his career path and primary goal to develop and commercialize his first product, a fully-biodegradable drug-eluting stent he calls the PowerStent® Absorb (see insert).  This technology combines three especially innovations:  a unique balloon-expandable stent design (PowerStent®), a bioabsorbable nanoparticle composition (BioDe®), and a formulation of two commercially-available anti-restenosis drugs (Combo®).

Stent

About the Subject

Dr. Wu received his clinical education in China and research training in the USA. In 1988, he graduated with an MD from the prestigious Linyli Medical School and completed a fellowship in clinical cardiology at the Tonji Medical University.  In 1993, presented with an opportunity to travel to the US, he uprooted to accept a position as visiting scholar, and ultimately post-doctoral fellow,in Jeffrey Isner’s lab at St. Elizabeth Hospital (Tufts University) and the Beth Israel Medical Center (Harvard Medical).  There he investigated the biology of stenosis, and directed sponsored research projects to evaluate the safety and efficacy of the latest commercially-developed drug-coated stents (DES) in animals.

After  a decade in academia, Dr. Wu made the successful transition to industry and joined Nitromed Inc. as a Research Scientist.  His next stop was as a Research Director at Biomedical Research Models, Inc (2000-2006) where we met and collaborated on developing and characterizing macrovascular disease in an inbred, type 2 diabetic rat model.  After a 20 year career, and upon gaining additional qualification in Mechanical Engineering (Wentworth Institute), Business Administration (MIT), Clinical Research Affairs (Mass. Biotech Council), and Medical Device Regulatory Affairs (North Eastern Univ.), he was ready to take the entrepreneurial leap.  His first company, VasoTech would aim to re-engineer the clinical standards of stent design and drug delivery.

In 2007, Dr. Wu founded VasoTech, Inc. from inside his home garage. Less than a year later, VasoTech received a $1.5M SBIR fast-track grant award from the NIH.  With funding, VasoTech joined the newly announced M2D2 facility on the University of Massachusetts Lowell campus, and expanded operations in China.  With the support of one of his closest advisors, Dr. Stephen McCarthy and other research faculty, Dr. Wu was appointed as an adjunct faculty in the Dept. of BioMedical Engineering at the UMass/Lowell where he mentored a number of talented graduate students.  Dr. Wu is recognized as a senior reviewer on the NIH Bioengineering, Surgical Science and Technology Study Section, and Biomaterials, Delivery Systems and Nanotechnology Special Emphasis Panels servicing the  Small Business Innovation Research (SBIR) grant program.

Dr. Wu’s work at Vasotech is devoted to developing a 3rd generation of fully biodegradable DES coronary stents to solve two major complications associated with stenting, restenosis and late-stage thrombosis. Thusfar, his ideas have attracted well over $1.5 Million (USD) in Small Business Innovation Research (SBIR) grant awards from the National Institute of Diabetes and Digestive and Kidney Diseases, and $1million (USD) from China Innovative Talent Leadership Program.  Through his efforts VasoTech is well positioned to attract the strategic partnerships and venture capital investments necessary to translate his research through clinical stages of development both in China and the US.

The Interview

Kislauskis:  Please help our readers understand the current clinical approach to CAD.

Wu:  Most patients with advanced atherosclerosis diseases are at risk for occlusive coronary arterial disease and stroke. Consequently, it is recommended they undergo a percutaneous intervention (PCI); essentially, balloon angioplasty followed by instillation of one or more expandable metal stents. A properly expanded stent will dilate the vessel and increase blood flow to cardiac muscle tissue. Current 2nd generation drug-eluting-stents (DES) release drugs to inhibit the process of vascular remodeling leading to restenosis. Because the DES approach is remarkably successful and lowers the rate of restenosis to < 10%, DESs is now performed in 85% of the 2 million percutaneous coronary interventions (PCI) procedures annually in the U.S.

Kislauskis:  What is your impression of the recent 5 yr update of the FREEDOM trial comparing effectiveness of coronary artery bypass grafting (CABG) to PCI among diabetics? 1

Wu:  It makes perfect sense. There are other reports evaluating PCI in patients within high risk categories, including those with small diameter vessels, diabetes, and extensive, systemic vascular disease, showing unacceptably high rates of restenosis with bare metal stents (30%-60%) and DESs (6%-18%) 2-4.  We also know first-hand using an inbred rat strain that develops macrovascular disease 4 months after onset of spontaneous diabetes.  In our experiment model, just 4weeks following balloon-induced injury to the coratid artery (PTCA),  we observed 2x greater restenosis in female obese rats, and 4x greater stenosis in obese, diabetic rats  littermates (syndrome X) relative to the non-obese, non-diabetic littermates.  These results predicted that obesity (dyslipidemia) and diabetes (severe hyperglycemia) were major risk factors promoting the complication of restenosis (Wu and Kislauskis, unpublished).

Kislauskis: Can you tell our readers a bit more about the significance of restenosis and thrombosis and the concept behind your approach.

Wu: Two significant drawbacks to conventional PCI are the need for costly, long-term anti-platelet therapy; and having a metal artifact within the coronary vessel. In fact, once installed, the purpose of DES is to maintain patency and provide a scaffold until remodeling is complete, maybe 6 months.  The period of drug elution is typically shorter in duration.  In the event of restenosis, a second DES procedure is recommended and performed with satisfactory results.  However, leaving another metal artifact is problematic.

Most concerning to PCI patients, however, should be an increased risk of sudden death from heart attack from a clot (thrombosis) and tissue ischemia (myocardial infarction).  No available DES technology (eg. Cypher®or Taxus® DES) demonstrates any advantage over bare metal stents in this regard 5-7.  So the thinking is a metal artifact create an irregular vessel surface and micro-eddys in blood flow which ultimately result in late-stage thrombosis, particularly in patients who go off anti-their platelet therapy too soon 8.  Therefore and conceptually, by combining potent DES technology with a fully-biodegradable scaffold, designed to be absorbed fully into the tissue, likely will reduce the rate in-stent stenosis and prevents late-stage thrombosis.

Kislauskis: How did you come up with your unique polymer formulation?

Wu: It turns out that through a process of trial and error in the lab I was able to identify a biodegradable formulation which reduces the local inflammatory response common to all DES formulations while improving the stent’s radial strength.  With a stable drug delivery platform (BioDe®), the process of remodeling will contribute far less to restenosis.  Furthermore, and unlike all prior art, my BioDe® formulation can neutralize acidic intermediates generated during stent degradation that induce inflammation.  The combination of anti-restenosis drugs (Combo®) also is effective at inhibiting signaling pathways that contribute to restenosis.

Kislauskis:  How did you come to design the PowerStent®?

Wu: Again, a long process of trial and error, initially using computer applied design (CAD) principals I learned while earning attending a mechanical engineering certificate program at Wentworth Institute of Technology in Boston. Elements behind my concept for BioDe® came to me while I was involved in a home renovation project, working with grout.  Although the formulation is simple and may be duplicated, the process of manufacturing is complicated.

Kislauskis: So it’s your trade secret.

Wu: Absolutely.

Kislauskis: Can you summary its other advantages and your plans to commercialize the PowerStent®?

Wu: Preclinical, short duration (30 day) studies in porcine models with the PowerStent® Absorb deployed indicate that it will be non-inferior to the current metal DES and competing biodegradable stent technologies. Important functional attributes of the BioDe® polymer include better biocompatibility (less inflammatory), excellent radial strength, potent anti-restenosis activity, and a unique microporous surface that promotes integration into neointimal layer of stented vessel.  Ongoing and much longer duration studies may also support our contention that this design can reduce risks of late-stage in-stent thrombosis.

Kislauskis: What path and difficulties to you foresee in obtaining a regulatory approval to conduct clinical trials with the PowerStent® Absorb?

Wu:  FDA Guidance to commercialize conventional DES technology is available. Unfortunately, no guidance is published for a fully-biodegradable stent.  Therefore, I anticipate seeking advice from the regulatory bodies prior to petitioning for approval to perform clinical trials.  It will no doubt be a complicated process as this technology involves a novel drug combination (albeit FDA-approved drugs), and a novel formulation (albeit FDA-approved components), and a novel indwelling and bioabsorbable medical device (stent).  We are presently completing several required engineering studies for the final phase of pre-clinical safety and efficacy testing, in China. The goals are to obtain FDA pre-market and NDA approvals, and to receive a CE mark from major international markets including Europe and the BRICK nations.

Kislauskis: How will you commercialize this 3rd generation, fully-biodegradable stent?

Wu: There are likely 3 scenarios to complete development and commercialization.  One involves securing bridge funding from the NIH SBIR program, supplemented with angel financing to complete preclinical program. I project that a minimum of $6 Million (USD) will be required to complete regulatory approval and pivotal clinical trials.  Therefore, it is conceivable that a Series A round of equity financing from venture capitalists, in either US or China, will be required. A third scenario is to partner or sell the technology to a major player in this space to complete clinical testing and commercialization. Potential partners include Boston Scientific Company, J&J, etc. Any of these partners could facilitate the processes of regulatory approval, manufacturing, global distribution and marketing.  Discussions are underway with one such prospective partner and with several VC groups.

Kislauskis: What is its likely impact of this product on patient care and the field of interventional cardiology?

Wu: According to US statistics, approximately 14 million Americans suffer from CAD, and 500,000 people die from acute myocardial infarction. One million more survive but with a 1.5 to 15 times greater risk of mortality or morbidity than the rest of the population each year.  In the U.S., the annual health care costs of CAD are estimated to be in excess of $112 billion, and the estimated annual total direct cost associated with PCI with stents is over $2 billion.  I anticipate that our PowerStent® Absorb stent will be competitive in a marketplace estimated to be over $5 billion in 2010. Although CAD patients are the primary market, other related applications for our PowerStent Absorb technology include peripheral arteries, intracerebral vascular and small vessels which are also significant.

Kislauskis:  Thank you for your contribution to this site.  For more information about MMG, LLC and Dr. Wu’s technology please refer to his publications 9-13 or contact him directly at tiangenwu@yahoo.com.

REFERENCES

1.   Mark A. Hlatky, M.D. Compelling Evidence for Coronary-Bypass Surgery in Patients with Diabetes.   N Engl J Med 2012; 367:2437-2438.

2.  Stamler, J. (1989) Epidemiology.  Established major risk factors, and the primary prevention of coronary heart disease. In: Chatterjee K, Karliner J, Rapaport E, Cheitlin MD, Parmlee WW, Sheinman, M eds. Cardiology, Philadelphia Penn: JB Lippincott, 1991, 7.2-7.35. (volume 2).

3. Tanabe, K, Regar, E et al.  Sirolimus-eluting stent for treatment of in-stentrestenosis: One-year angiographic and intravascular ultrasound follow-up. J. Am Col.Cardi.   (2003) 41: 12A.

4. Grube, Eberhard;  Silber, Sigmund.  Six- and twelve-month results from a randomized, double-blind trial on a slow-release paclitaxel-eluting stent for de novo coronary lesions. Circulation 2003: 107, 38-42.

5.  Iakovou I, Schmidt T, Bonizzoni E, et al. Incidence, predictors, and outcome of thrombosis after successful implantation of drug-eluting stents. JAMA 2005;293:2126–2130.

6.  Ong AT, McFadden EP, Regar E, et al. Late angiographic stent thrombosis (LAST) events with drug-eluting stents. J Am Coll Cardiol 2005;45:2088–2092.

7. Wang F, Stouffer GA, Waxman S, et al. Late coronary stent thrombosis: Early vs late stent thrombosis in the stent era. Catheter Cardiovasc Interven 2002;55:142–147.

8. McFadden EP, Stabile E, Regar E, et al. Late thrombosis in drug-eluting coronary stents after discontinuation of antiplatelet therapy. Lancet 2004;364:1519–1521.

9. Ma X, Oyamada S, Wu T, Robich MP, Wu H, Wang X, Buchholz B, McCarthy S, Bianchi CF, Sellke FW, Laham R. In vitro and in vivo degradation of poly(D, L-lactide-co-glycolide)/amorphous calcium phosphate copolymer coated on metal stents. J Biomed Mater Res A. 2011 Mar 15;96(4):632-8. doi: 10.1002/jbm.a.33016. Epub 2011 Jan 25.

10. Oyamada S, Ma X, Wu T, Robich MP, Wu H, Wang X, Buchholz B, McCarthy S, Bianchi CF, Sellke FW, Laham R. Trans-iliac rat aorta stenting: a novel high throughput preclinical stent model for restenosis and thrombosis. J Surg Res. 2011 Mar;166(1):e91-5. Erratum in: J Surg Res. 2012 May 1;174(1):184.

11. Ma X, Oyamada S, Gao F, Wu T, Robich MP, Wu H, Wang X, Buchholz B, McCarthy S, Gu Z, Bianchi CF, Sellke FW, Laham R Paclitaxel/sirolimus combination coated drug-eluting stent: in vitro and in vivo drug release studies. J Pharm Biomed Anal. 2011 Mar 25;54(4):807-11. Erratum in: J Pharm Biomed Anal. 2012 Feb 5;59:217.

12. Ma X, Wu T, Robich MP, Wang X, Wu H, Buchholz B, McCarthy S. Drug-eluting stents. Int J Clin Exp Med. 2010 Jul 15;3(3):192-201.

Other articles related to this subject were published in this Open Access OnlIne Scientific Journal:

Lev-Ari, A. (2012aa). Renal Sympathetic Denervation: Updates on the State of Medicine

http://pharmaceuticalintelligence.com/2012/12/31/renal-sympathetic-denervation-updates-on-the-state-of-medicine/

 

Lev-Ari, A. (2012U). Imbalance of Autonomic Tone: The Promise of Intravascular Stimulation of Autonomics

http://pharmaceuticalintelligence.com/2012/09/02/imbalance-of-autonomic-tone-the-promise-of-intravascular-stimulation-of-autonomics/

Lev-Ari, A. (2012R). Coronary Artery Disease – Medical Devices Solutions: From First-In-Man Stent Implantation, via Medical Ethical Dilemmas to Drug Eluting Stents http://pharmaceuticalintelligence.com/2012/08/13/coronary-artery-disease-medical-devices-solutions-from-first-in-man-stent-implantation-via-medical-ethical-dilemmas-to-drug-eluting-stents/

 

Lev-Ari, A. (2012K). Percutaneous Endocardial Ablation of Scar-Related Ventricular Tachycardia

http://pharmaceuticalintelligence.com/2012/07/18/percutaneous-endocardial-ablation-of-scar-related-ventricular-tachycardia/

 

Lev-Ari, A. (2012C). Treatment of Refractory Hypertension via Percutaneous Renal Denervation

http://pharmaceuticalintelligence.com/2012/06/13/treatment-of-refractory-hypertension-via-percutaneous-renal-denervation/

Lev-Ari, A. (2012D). Competition in the Ecosystem of Medical Devices in Cardiac and Vascular Repair: Heart Valves, Stents, Catheterization Tools and Kits for Open Heart and Minimally Invasive Surgery (MIS)

http://pharmaceuticalintelligence.com/2012/06/22/competition-in-the-ecosystem-of-medical-devices-in-cardiac-and-vascular-repair-heart-valves-stents-catheterization-tools-and-kits-for-open-heart-and-minimally-invasive-surgery-mis/

Lev-Ari, A. (2012E). Executive Compensation and Comparator Group Definition in the Cardiac and Vascular Medical Devices Sector: A Bright Future for Edwards Lifesciences Corporation in the Transcatheter Heart Valve Replacement Market

http://pharmaceuticalintelligence.com/2012/06/19/executive-compensation-and-comparator-group-definition-in-the-cardiac-and-vascular-medical-devices-sector-a-bright-future-for-edwards-lifesciences-corporation-in-the-transcatheter-heart-valve-replace/

 

Lev-Ari, A. (2012F). Global Supplier Strategy for Market Penetration & Partnership Options (Niche Suppliers vs. National Leaders) in the Massachusetts Cardiology & Vascular Surgery Tools and Devices Market for Cardiac Operating Rooms and Angioplasty Suites

http://pharmaceuticalintelligence.com/2012/06/22/global-supplier-strategy-for-market-penetration-partnership-options-niche-suppliers-vs-national-leaders-in-the-massachusetts-cardiology-vascular-surgery-tools-and-devices-market-for-car/

 

Lev-Ari, A. (2012G).  Heart Remodeling by Design: Implantable Synchronized Cardiac Assist Device: Abiomed’s Symphony

http://pharmaceuticalintelligence.com/2012/07/23/heart-remodeling-by-design-implantable-synchronized-cardiac-assist-device-abiomeds-symphony/

 

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PCI Outcomes, Increased Ischemic Risk associated with Elevated Plasma Fibrinogen not Platelet Reactivity

Reporter: Aviva Lev-Ari, PhD, RN

Article ID #13: PCI Outcomes, Increased Ischemic Risk associated with Elevated Plasma Fibrinogen not Platelet Reactivity. Published on 1/10/2013

WordCloud Image Produced by Adam Tubman

 

Q&A Session between Dr. Michael Ward and Dr. Larry Bernstein presented for in our Research Category on 

Interviews with Scientific Leaders

Primary research:

Ang L, et al “Elevated plasma fibrinogen rather than residual platelet reactivity after clopidogrel pre-treatment is associated with an increased ischemic risk during elective percutaneous coronary intervention” J Am Coll Cardiol2013; 61: 23-34.

 

Question by DR. MICHAEL WARD

How ironic that an old diagnostic parameter should
reappear in the limelight of diagnostic predictors.

Of course, decades ago, doctors asked for “sed rates”, seeking to know if red cells, thought to be bound to fibrinogen, settled faster in a patient compared to a control subject’s blood. Fibrinogen has always been a diagnostic number in evaluating inflammatory results.

However, the diagnostic world, like the worlds of pharmaceuticals, medical devices, biologics, and other industries, always seek the ‘new kid on the block’ to differentiate themselves from the rest of the pack in the
marketplace.

So there was a binge (and still is) to seek new and exotic blood proteins that are surrogate markers for specific diagnoses or prognoses.

That is the irony, that in this case at least, fibrinogen has come full circle. Biology works in mysterious ways.

Answer by Dr. Larry Bernstein, MD, FCAP

Dear Dr. M.  Ward:

Doctors asked for “sed rates”, seeking to know if red cells, thought to be bound to fibrinogen, settled faster in a patient compared to a control
subject’s blood. Fibrinogen has always been a diagnostic number in evaluating inflammatory results.

You are quite right that physicians used “sed rates” as a measure of inflammation, and more in Lupus Erythematosis, Rheumatoid Arthritis, Nephritides, Systemic Sclerosis, and so forth.  The “sed rate” was not a part of the thinking about CVD, and PCI didn’t exist.  Recently, MI post-PCI has been defined as a type (NSTEMI?).

Yes. In principle, the sed rate is related to fibrinogen and red-cell aggregation.  I am not prepared to accept that a platelet count over 400,000 would make no contribution, even if many of the PCI related infarcts are within a range of 150-300,000.  I don’t know how much power there is in the discussion.  The role of tissue factor (plaque), and of platelets in hemostasis is undeniable.

The industry does look for every opportunity to seize on promising biomarkers.  The coagulation assays developed at Dade-Behring (Dade, Dupont Division; then Dade) were far better and more explanatory that the “sed rate”.  The sed rate measurement requires that you set up graduated tubes to watch the rate of sedimentation.  It is not a walkaway procedure.  Industry has been so good at introducing automation that led to high volume efficiency, that this led to the only part of hospital operations that had good accounting measures.  The long trip to reducing personnel, but of course the profiles were a piece of cake.  I continually reorganized to carve out services for immunology and toxicology, which took longer to get automated.

The only use for sed rate now is for Temporal Thrombosis (?).

In the early days Yale NH Hospital had some 5 Perkin Elmer HPLCs to measure calcium.  Electrophoretic separation of isoenzymes was not helpful for managing patients.  The procedure was run batchwise once a day.  I was the first in CT to be running the immunoassay three times a day on the Roche COBAS Bio CFA., and Dupont put it on the ‘aca’.  A med tech could run it at 3 am  at Detroit Receiving, Bellevue, or Cook County, when the phone didn’t stop ringing for STAT results.

Physicians had expectations too.  So we had the progression from AST, LDH, and CK to isoenzyme MBCK, and then there were the cancer biomarkers – CEA, CA-125, PSA, with much to be discussed.

 

Q&A is derived from the following Article in

MedPage Today

Published: January 07, 2013

Fibrinogen Level Tied to Poorer PCI Outcomes

By Todd Neale, Senior Staff Writer, MedPage Today

Published: January 07, 2013

Reviewed by Robert Jasmer, MD; Associate Clinical Professor of Medicine, University of California, San Francisco and Dorothy Caputo, MA, BSN, RN, Nurse Planner

An elevated serum fibrinogen level predicted worse short-term ischemic outcomes among patients undergoing elective percutaneous coronary intervention after pretreatment with clopidogrel, researchers found.

Significantly higher levels of fibrinogen were seen in patients with periprocedural myocardial infarction (MI) defined by either creatine kinase-myocardial band (CK-MB) or troponin (P<0.02 for both), according to Ehtisham Mahmud, MD, of the University of California, San Diego, and colleagues.

Those relationships remained consistent after adjustment for several factors, including platelet function, which was not itself associated with periprocedural MI, the researchers reported in the Jan. 8 issue of the Journal of the American College of Cardiology.

“The results of the current study suggest that an elevated fibrinogen level…is related to significant platelet cross-linking and thrombus formation independent of residual P2Y12 receptor-mediated platelet activity during clopidogrel therapy,” they wrote.

Higher risk of ischemic cardiovascular events has been observed with both high platelet reactivity after thienopyridine treatment and elevated serum fibrinogen.

“As an acute phase reactant involved in the final common pathway of the coagulation cascade and essential component of platelet cross-linking in thrombus formation, fibrinogen possesses a clear biological mechanism for its adverse cardiovascular effects,” Mahmud and colleagues wrote.

In fact, high levels of serum fibrinogen have been shown to contribute to high platelet reactivity during clopidogrel treatment, resulting in uncertainty about whether insufficient platelet inhibition and elevated fibrinogen levels are independent or interactive risk factors for ischemic events.

To explore the issue, the researchers looked at data from 189 patients undergoing elective PCI who were pretreated with clopidogrel, defined as 75 mg daily for at least 7 days or a 600-mg bolus at least 12 hours before study enrollment. The mean age of the patients was 63.8 and most (74.1%) were male.

Nearly two-thirds (63%) had undergone a previous PCI, and 18% had undergone revascularization with coronary artery bypass grafting (CABG).

Baseline platelet function was measured using the VerifyNow P2Y12 assay. Markers of ischemic myocardial injury, including troponin and CK-MB, were measured every 8 hours after PCI until hospital discharge.

Periprocedural MI defined by troponin I or T occurred in 13.9% of patients. Those who had an MI had significantly higher levels of fibrinogen (363.1 versus 309.1 mg/dL, P=0.017).

The rate of CK-MB-defined periprocedural MI was 5.8%. Patients with that outcome also had elevated levels of fibrinogen (403.4 versus 313.5 mg/dL, P=0.007).

Both differences remained significant after multivariate adjustment that accounted for platelet function and other inflammatory markers.

The researchers found that a fibrinogen level of 345 mg/dL or higher — a cutoff identified as having optimal combined sensitivity and specificity for CK-MB-defined periprocedural MI — was associated with periprocedural MI defined by either troponin or CK-MB (P<0.04 for both).

Those relationships were stronger when systemic inflammation was low (C-reactive protein ≤0.5 mg/dL).

The platelet reactivity measurements were not associated with either definition of periprocedural MI, which is inconsistent with the findings from several smaller studies. The authors noted, however, that “the significance of these negative findings may be limited due to inadequate study power.”

In discussing the limitations of the study, the researchers pointed out that “the findings … do not provide insight into whether the relationship between high platelet reactivity and ischemic cardiovascular events demonstrated in previous studies is a direct one or mediated through the effect of serum fibrinogen.”

To get to the bottom of that, they wrote, “future studies relating platelet reactivity and adverse cardiac events should measure baseline fibrinogen.”

Mahmud has received clinical trial support from Accumetrics, Eli Lilly, and sanofi-aventis, and is on the speakers bureau for Medtronic. One of his co-authors is a consultant for Abbott Vascular, Boston Scientific, St. Jude Medical, Medtronic, and sanofi-aventis.

From the American Heart Association:

Todd Neale

Senior Staff Writer

Todd Neale, MedPage Today Staff Writer, got his start in journalism at Audubon Magazine and made a stop in directory publishing before landing at MedPage Today. He received a B.S. in biology from the University of Massachusetts Amherst and an M.A. in journalism from the Science, Health, and Environmental Reporting program at New York University.

SOURCE:

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Reporter: Aviva Lev-Ari, PhD, RN

Tool Identifies Risk in Stenting ACS Patients

By Todd Neale, Senior Staff Writer, MedPage Today

Published: November 19, 2012
Reviewed by Dori F. Zaleznik, MD; Associate Clinical Professor of Medicine, Harvard Medical School, Boston and Dorothy Caputo, MA, BSN, RN, Nurse Planner

A new, easy-to-calculate risk score developed for patients with non-ST-segment elevation acute coronary syndromes (ACS) undergoing percutaneous coronary intervention (PCI) had better prognostic accuracy than other widely used risk scores, researchers found.

The ACUITY-PCI risk score includes six variables — insulin-treated diabetes, renal insufficiency, baseline cardiac biomarker elevation or ST-segment deviation, presence of a bifurcation lesion, small vessel/diffuse coronary artery disease, and extent of coronary artery disease, according to Gregg Stone, MD, of Columbia University Medical Center in New York City, and colleagues.

The 1-year rate of death or MI significantly increased from 5.3% in the lowest risk tertile to 9.1% in the middle tertile to 19% in the highest tertile (P<0.001), the researchers reported in the November issue of JACC: Cardiovascular Interventions.

Discrimination and calibration were greater with the ACUITY-PCI score than with other established scores.

“Although the TIMI and the GRACE scores have been shown to be valuable prognostic tools at the time of hospital admission for selecting pharmacological strategies and identifying those patients most likely to benefit from an invasive strategy, they have not been optimized for patients undergoing PCI and, thus, have relatively poor prognostic power to further risk stratify acute coronary syndrome patients undergoing PCI,” Stone and colleagues wrote.

“The ACUITY-PCI score is therefore intended to supplement the TIMI and GRACE scores when an invasive strategy has been undertaken and PCI is being considered.”

The researchers created the risk score using data from 1,692 patients enrolled in the angiographic substudy of the ACUITY trial, which was a comparison of heparin plus a glycoprotein IIb/IIIa inhibitor, bivalirudin (Angiomax) plus a glycoprotein IIb/IIIa inhibitor, or bivalirudin alone in patients with ACS undergoing an early invasive strategy. They then validated the score using another 846 patients from the same study.

Multivariate analysis revealed six variables that were significantly associated with 1-year mortality and MI and were included in the score. The researchers assigned points based on the strength of the predictor:

  • Insulin-treated diabetes (12 points)
  • Renal insufficiency (12 points)
  • Baseline cardiac biomarker elevation or ST-segment deviation (8 points)
  • Bifurcation lesion (4 points)
  • Small vessel/diffuse coronary artery disease (2 points)
  • Extent of coronary artery disease (1 point for each 10 mm of disease)

The C-statistic for the risk score — a measure of discrimination — was 0.67 in the derivation cohort and 0.70 in the validation cohort. In the validation cohort, the chi-square statistic for calibration was 6.2 and the index of separation was 0.44.

All of those values were better than those seen for four other established risk scores — TIMI, GRACE, SYNTAX, and Clinical SYNTAX. In addition, the net reclassification improvement with the new score ranged from 9% to 38% and the integrated discrimination index varied from 1.9% to 2.7%.

The researchers noted that the ACUITY-PCI score also was a good predictor of 1-year definite or probable stent thrombosis, with a C-statistic of 0.72.

In another study in the same journal, George Dangas, MD, PhD, of Mount Sinai Medical Center in New York City, and colleagues — including Stone — reported on the development of a risk score specifically for stent thrombosis in patients with ACS undergoing PCI.

The study included 6,139 patients from the HORIZONS-AMI and ACUITY trials, which included those with ST-segment elevation MI (STEMI) in the former trial and those with non-STEMI and unstable angina in the latter. The researchers used 4,093 patients for the derivation cohort and 2,046 for the validation cohort.

The risk score included 10 variables that were significantly associated with the risk of Academic Research Consortium-defined definite or probable stent thrombosis at 1 year:

  • Type of acute coronary syndrome (4 points for STEMI, 2 points for non-ST-segment elevation ACS with ST deviation, and 1 point for non-ST-segment elevation ACS without ST changes)
  • Current smoking (1 point)
  • Insulin-dependent diabetes (2 points)
  • Prior PCI (1 point)
  • Baseline platelet count (1 point for 250 to 400 K/µL and 2 points for more than 400 K/µL)
  • Absence of pre-PCI heparin therapy (1 point)
  • Aneurysmal/ulcerated lesion (2 points)
  • Baseline TIMI flow grade 0/1 (1 point)
  • Final TIMI flow grade less than 3 (1 point)
  • Number of treated vessels (1 point for two vessels and 2 points for three vessels)

Scores from 1 to 6 are considered low risk, 7 to 9 are intermediate risk, and 10 or higher are high risk.

The rates of stent thrombosis at 1 year were 1.36%, 3.06%, and 9.18% across the three risk tertiles in the derivation cohort (P<0.001 for trend), with a similar trend seen in the validation cohort.

The C-statistics were 0.67 in the derivation cohort and 0.66 in the validation cohort. Performance was comparable for events occurring both early (within the first 30 days) and late (from 1 month to 1 year).

“We believe that the development and initial validation of this stent thrombosis risk score can be a useful tool for both clinical practice and future clinical investigation (future analyses of trials or registries), as it can be a simple way to risk stratify patients immediately following a procedure,” Dangas and colleagues wrote. “The risk score could also be used in the informed consent process to better inform patients of their individual risk of stent thrombosis.”

But Ron Waksman, MD, and Israel Barbash, MD, of MedStar Washington Hospital Center in Washington, D.C., noted some limitations of the tool, including the pooling of different types of patients, the exclusion of important variables associated with stent thrombosis risk, and the use of mostly first-generation drug-eluting stents in the trials.

“It is imperative that the user of such a prediction tool be aware of its capabilities and performance, as well as its limitations, in various clinical scenarios,” they wrote in an accompanying editorial.

“A newly developed risk score for stent thrombosis should be robust and should be tested across broad study populations, stents, and antiplatelet regimens. A new model should also be validated in a setting different from the one in which it was derived,” they wrote. “Unfortunately, this is not the case with the newly proposed model.”

“Until such an encompassing tool is developed and validated,” they wrote, “one should rely on the known stent thrombosis risk factors and tailor an appropriate treatment for each patient.”

The ACUITY trial was funded by The Medicines Company and Nycomed.

Stone has served as a consultant to Abbott Vascular, Boston Scientific, Medtronic, and The Medicines Company. His co-authors reported relationships with Abbott, Regado, Ortho McNeil, Janssen, Merck, Maya Medical, AstraZeneca, Sanofi/Bristol-Myers Squibb, Eli Lilly, and Daiichi Sankyo.

The HORIZONS-AMI trial was supported by the Cardiovascular Research Foundation, with grant support from Boston Scientific and The Medicines Company.

Dangas has received speaker honoraria from AstraZeneca, Bristol-Myers Squibb, The Medicines Company, sanofi-aventis, and Abbott Vascular. His co-authors reported relationships with sanofi-aventis, The Medicines Company, Abbott Vascular, Bristol-Myers Squibb, Cordis, AstraZeneca, Daiichi Sankyo, Eli Lilly, Maquet, Roche, Boehringer Ingelheim, Liposcience, Merck, Pozen, Gilead Sciences, WebMD, the NIH, Pfizer, Johnson & Johnson, Schering-Plough, Merck Sharpe and Dohme, GlaxoSmithKline, Regado Biosciences, Boston Scientific, and Bristol-Myers Squibb/Sanofi.

Waksman and Barbash reported that they had no conflicts of interest.

From the American Heart Association:

Primary source: JACC: Cardiovascular Interventions
Source reference:
Palmerini T, et al “A new score for risk stratification of patients with acute coronary syndromes undergoing percutaneous coronary intervention: the ACUITY-PCI (Acute Catheterization and Urgent Intervention Triage Strategy-Percutaneous Coronary Intervention) risk score” JACC Cardiovasc Interv 2012; 5: 1108-1116.

Additional source: JACC: Cardiovascular Interventions
Source reference:
Dangas G, et al “Development and validation of a stent thrombosis risk score in patients with acute coronary syndromes” JACC Cardiovasc Interv 2012; 5: 1097-1105.

Additional source: JACC: Cardiovascular Interventions
Source reference:
Waksman R, Barbash I “The appropriate use of risk scores” JACC Cardiovasc Interv 2012; 5: 1106-1107.

Todd Neale

Senior Staff Writer

Todd Neale, MedPage Today Staff Writer, got his start in journalism at Audubon Magazine and made a stop in directory publishing before landing at MedPage Today. He received a B.S. in biology from the University of Massachusetts Amherst and an M.A. in journalism from the Science, Health, and Environmental Reporting program at New York University. He is based atMedPage Today headquarters in Little Falls, N.J.

SOURCE:

http://www.medpagetoday.com/Cardiology/AcuteCoronarySyndrome/36010

Read Full Post »

UPDATED: PLATO Trial on ACS: BRILINTA (ticagrelor) better than Plavix® (clopidogrel bisulfate): Lowering chances of having another heart attack

Reporter: Aviva Lev-Ari, PhD, RN

 

UPDATED on 9/1/2019

Extended DAPT with Brilinta: No Benefit for Stable CAD in T2D

Substudy in those with prior PCI might identify group where bleeding tradeoff is worthwhile

PARIS — Ticagrelor (Brilinta) as part of a dual antiplatelet therapy (DAPT) regimen didn’t improve net outcomes for stable coronary artery disease (CAD) among type 2 diabetes patients, except perhaps in the setting of percutaneous coronary intervention (PCI), the THEMIS trial showed.

Adding the potent antiplatelet agent to aspirin reduced cardiovascular (CV) death, myocardial infarction (MI), or stroke (7.7% vs 8.5%, HR 0.90, 95% CI 0.81-0.99), reported Deepak Bhatt, MD, MPH, of Brigham and Women’s Hospital and Harvard Medical School in Boston, at the European Society of Cardiology (ESC) congress and online in the New England Journal of Medicine.

But it also increased

  • TIMI major bleeding (2.2% vs 1.0%, HR 2.32, 95% CI 1.82-2.94) and
  • intracranial hemorrhage (0.7% vs 0.5%, HR 1.71, 95% CI 1.18- 2.48) over aspirin alone, albeit
  • without more fatal bleeding (0.2% vs 0.1%, P=0.11).

The combined effect was neutral for the exploratory composite outcome of “irreversible harm” (death from any cause, MI, stroke, fatal bleeding, or intracranial hemorrhage 10.1% vs 10.8%, HR 0.93, 95% CI 0.86-1.02).

ESC session study discussant Colin Baigent, MD, of Oxford University in England, actually calculated 12 major bleeds for every eight events prevented.

“This is a consistent story: when we add an antiplatelet agent for risk reduction, we increase the risk of bleeding,” noted Richard Kovacs, MD, of Indiana University in Indianapolis and president of the American College of Cardiology.

THEMIS is the final part of a largely-disappointing PARTHENON development program for ticagrelor, he noted. “It hasn’t changed practice. …Will the main THEMIS trial change clinical practice? In my opinion, no.”

SOURCE

https://www.medpagetoday.com/meetingcoverage/esc/81925?xid=nl_mpt_ACC_Reporter_2019-09-01&eun=g5099207d2r

 

UPDATED on 10/4/2016

Soriot’s $3.5B Brilinta dream is dashed by yet another big trial flop for AstraZeneca

by john carroll
October 4, 2016 09:00 AM EDT
Updated: 09:33 AM

Brilinta, the drug failed to demonstrate a benefit over generic Plavix (clopidogrel) for peripheral artery disease. Back in March, the heart drug flopped in a large stroke study, unable to prove that it could beat aspirin. And Soriot can chalk up those expensive studies to proving Brilinta’s serious deficiencies.

“We don’t believe the goal of $3.5 billion is attainable. I think it would be unrealistic to believe that,” Ludovic Helfgott, head of AstraZeneca’s Brilinta business, told Reuters.

Brilinta brought in a total of $619 million last year after disappointing analysts repeatedly with lower-than-expected quarterly revenue.

Heart studies aren’t cheap. AstraZeneca recruited 13,500 patients for the EUCLID study, and it had enrolled close to that number for the earlier SOCRATES trial.

SOURCE

http://endpts.com/soriots-3-5b-brilinta-dream-is-dashed-by-yet-another-big-trial-flop-for-astrazeneca/?utm_medium=email&utm_campaign=75%20Dinner%20with%20Brent&utm_content=75%20Dinner%20with%20Brent+CID_8008d3b4f16d90576238cceef624d211&utm_source=ENDPOINTS%20emails&utm_term=Soriots%2035B%20Brilinta%20dream%20is%20dashed%20by%20yet%20another%20big%20trial%20flop%20for%20AstraZeneca

UPDATED on 9/4/2014

Prehospital Ticagrelor in ST-Segment Elevation Myocardial Infarction

Gilles Montalescot, M.D., Ph.D., Arnoud W. van ‘t Hof, M.D., Ph.D., Frédéric Lapostolle, M.D., Ph.D., Johanne Silvain, M.D., Ph.D., Jens Flensted Lassen, M.D., Ph.D., Leonardo Bolognese, M.D., Warren J. Cantor, M.D., Ángel Cequier, M.D., Ph.D., Mohamed Chettibi, M.D., Ph.D., Shaun G. Goodman, M.D., Christopher J. Hammett, M.B., Ch.B., M.D., Kurt Huber, M.D., Magnus Janzon, M.D., Ph.D., Béla Merkely, M.D., Ph.D., Robert F. Storey, M.D., D.M., Uwe Zeymer, M.D., Olivier Stibbe, M.D., Patrick Ecollan, M.D., Wim M.J.M. Heutz, M.D., Eva Swahn, M.D., Ph.D., Jean-Philippe Collet, M.D., Ph.D., Frank F. Willems, M.D., Ph.D., Caroline Baradat, M.Sc., Muriel Licour, M.Sc., Anne Tsatsaris, M.D., Eric Vicaut, M.D., Ph.D., and Christian W. Hamm, M.D., Ph.D. for the ATLANTIC Investigators

September 1, 2014DOI: 10.1056/NEJMoa1407024

BACKGROUND

The direct-acting platelet P2Y12 receptor antagonist ticagrelor can reduce the incidence of major adverse cardiovascular events when administered at hospital admission to patients with ST-segment elevation myocardial infarction (STEMI). Whether prehospital administration of ticagrelor can improve coronary reperfusion and the clinical outcome is unknown.

METHODS

We conducted an international, multicenter, randomized, double-blind study involving 1862 patients with ongoing STEMI of less than 6 hours’ duration, comparing prehospital (in the ambulance) versus in-hospital (in the catheterization laboratory) treatment with ticagrelor. The coprimary end points were the proportion of patients who did not have a 70% or greater resolution of ST-segment elevation before percutaneous coronary intervention (PCI) and the proportion of patients who did not have Thrombolysis in Myocardial Infarction flow grade 3 in the infarct-related artery at initial angiography. Secondary end points included the rates of major adverse cardiovascular events and definite stent thrombosis at 30 days.

RESULTS

The median time from randomization to angiography was 48 minutes, and the median time difference between the two treatment strategies was 31 minutes. The two coprimary end points did not differ significantly between the prehospital and in-hospital groups. The absence of ST-segment elevation resolution of 70% or greater after PCI (a secondary end point) was reported for 42.5% and 47.5% of the patients, respectively. The rates of major adverse cardiovascular events did not differ significantly between the two study groups. The rates of definite stent thrombosis were lower in the prehospital group than in the in-hospital group (0% vs. 0.8% in the first 24 hours; 0.2% vs. 1.2% at 30 days). Rates of major bleeding events were low and virtually identical in the two groups, regardless of the bleeding definition used.

CONCLUSIONS

Prehospital administration of ticagrelor in patients with acute STEMI appeared to be safe but did not improve pre-PCI coronary reperfusion. (Funded by AstraZeneca; ATLANTIC ClinicalTrials.gov number, NCT01347580.)

SOURCE

http://www.nejm.org/doi/full/10.1056/NEJMoa1407024?query=TOC

 

 

UPDATED on 2/7/2014

PLATO Controversy Hits the Wall Street Journal

February 05, 2014

NEW YORK, NY – The controversy surrounding the PLATOtrial of ticagrelor (Brilinta, AstraZeneca) continues unabated, according to a story published in the Wall Street Journal. Specifically, a sealed complaint filed in US district court in the District of Columbia by a researcher contends that the cardiovascular events in the study “may have been manipulated” [1].

Dr Victor Serebruany (HeartDrug Research Laboratories, Johns Hopkins University, Towson, MD), who has long been a thorn in the side of AstraZeneca and the PLATO investigators, filed the complaint under the False Claims Act, reports theWall Street Journal. The Journal notes that the US attorney’s office in Washington, DC, has contacted Serebruany and is currently investigating the clinical trial.As reported by heartwirein October 2013, the US Department of Justice issued a civil investigative demand from its civil division “seeking documents and information regarding PLATO.” AstraZeneca is complying with the request.

First reported by heart wirein 2009 , the PLATO trial was a positive study involving more 18 000 patients from 43 countries. PLATO investigators, led by Dr Lars Wallentin (Uppsala Clinical Research Center, Sweden), showed that treating acute coronary syndrome patients with ticagrelor significantly reduced the rate of MI, stroke, and cardiovascular death compared with patients taking clopidogrel. Results were presented at the European Society of Cardiology 2009 Congress and reported in the New England Journal of Medicine.

PLATO has been dogged by questions, including prior to approval. In the sealed complaint, Serebruany takes issue with a number of things, many of which have been reported previously. He alleges that the

  • number of clinical events among those taking clopidogrel was high compared with other studies, pointing out that the rate of all-cause death was 5.9% among clopidogrel-treated patients—nearly twice as high as earlier studies. In addition,
  • the sealed complaint documents the geographic discrepancies in the trial, noting there was a trend toward worse outcomes with ticagrelor at North American sites.The complaint also alleges that
  • an initial count of clinical events suggested the two drugs were equivalent, but adjudication by the Duke Clinical Research Institute attributed another 45 MIs to the clopidogrel group, which tipped the results in favor of ticagrelor. Other questions raised about the study include
  • site monitoring and timing of clinical events. Serebruany also alleges that
  • the trial may have unintentionally been unblinded because of the shape of clopidogrel’s “split capsules,” which would have enabled doctors and nurses to know which drug patients received.

AstraZeneca rebutted these issues, telling the Journal that it is cooperating with the government. It said it is confident in the integrity of the trial and noted the overall study showed the superiority of ticagrelor over clopidogrel. There is no evidence the trial was unblinded and researchers used the same standards when qualifying all clinical events, including MIs, they noted. In addition, the company said it is not possible to compare event rates with clopidogrel in PLATO with other studies because the patient populations differ.

The Journal reports that Serebruany became embroiled in the controversy when asked by the FDA‘s Dr Thomas Marciniak to advise the agency about the PLATO data in 2010. Marciniak, who led the FDA’s review of PLATO, called AstraZeneca’s submission on serious adverse events the “worst submission” he ever encountered. According to the submission, he noted, 12 patients reported their own deaths by telephone. Before approving ticagrelor, the FDA requested an additional analysis of PLATO, and it was eventually approved in the US in July 2011. Ticagrelor was approved in Europe in December 2010 and is authorized for use in more than 100 countries.

The Journal called Serebruany an expert in the antiplatelet field but said he is a “controversial figure,” partly because of his financial ties to industry and repeated criticisms of new drug approvals. Through HeartDrug Research, Serebruany has worked on prasugrel (Effient, Lily/Daiichi-Sankyo), a competing antiplatelet agent, but has also done work for AstraZeneca.

REFERENCE

Burton TM. Doctor challenges testing of AstraZeneca’s Brilinta. Wall Street Journal, February 2, 2014. Available here.

SOURCE

http://www.medscape.com/viewarticle/820236?nlid=47583_1984&src=wnl_edit_medn_card&uac=93761AJ&spon=2

UPDATED 3/28/2013

How AstraZeneca Will Use A Diagnostic To Market Its Blood Thinner

by Matthew Harper, Forbes Staff on 3/21/2013

Earlier today I wrote about how AstraZeneca is telling investors that its blood-thinner Brilinta, used to prevent second heart attacks, could be a multi-billion dollar drug, at least twice as big as Wall Street analysts expect. So far the drug has been a disappointment.

I wrote:

Another key data point Astra presented was that blood levels of troponin, a muscle protein released by the heart during a heart attack, predict which patients get the most benefit from Brilinta. This data is not in AstraZeneca’s label, but a spokeswoman said that she believed it would be something the company can market to doctors.

via Can Pascal Soriot Turn Around AstraZeneca? It May Come Down To One Drug – Forbes.

But will the Food and Drug Administration allow Astra to tell doctors that? Stratification using troponin is not in Brilinta’s FDA-approved label, and off-label promotion is illegal. But Ferguson says that communications about troponin will be allowed because all patients with high troponin are patients who would be included in the FDA-approved indication. He confirms that use of troponin testing will be part of the new marketing plan for Brilinta.

SOURCE:

http://www.forbes.com/sites/matthewherper/2013/03/21/how-astrazeneca-will-use-a-diagnostic-to-market-its-blood-thinner/

Can Pascal Soriot Turn Around AstraZeneca? It May Come Down To One Drug

by Matthew Herper, Forbes Staff on 3/21/2013

This morning in New York, new AstraZeneca chief executive Pascal Soriot is telling investors how he is going to turn around the company that has had the absolute worst track record in research and development among any big pharmaceutical firm. The plan is fairly wide-ranging and involves a lot of the steps one might expect:

  • new layoffs (2,300 jobs);
  • a re-focusing of research and development on three areas: heart disease and diabetes; oncology; and respiratory and inflammation;
  • new R&D initiatives involving Moderna, a biotech company, and the Karolinska Instutet;
  • moving the company’s headquarters to its R&D hub in Cambridge, U.K.;
  • re-focusing on emerging markets, where AZ already gets $6 billion in sales, especially China.

But the short-term key to delivering on his promises today seems to come down to a single drug: Brilinta, the Plavix competitor thatAstraZeneca introduced in 2011 which has so far disappointed, generating  just $324 $89 million in global sales last year. This is a medicine to prevent heart attacks and strokes in patients who suffer acute coronary syndrome, the condition that occurs after a heart attack or serious heart-related chest pain. It works by preventing the formation of blood clots.

Plavix was the second biggest drug in the world, with $6 billion in annual sales, but it is now generic. The conventional wisdom is that it will be difficult to compete with cheap generics. Brilinta is actually trailing Effient, a similar medicine from Eli Lilly, in usage. Wall Street consensus currently sees Brilinta growing to become a moderate-sized drug in 2018, with $1.3 billion in annual sales. But AstraZeneca is saying that it thinks Brilinta can be a multi-billion dollar product. Astra has confirmed that this means Brilinta will have to surpass Effient. The newer drugs also cause more bleeding than Plavix.

What is the company’s argument? In his presentation today, Paul Hudson, Astra’s Executive Vice President, North America, said that the key would be focusing on one key fact: Brilinta reduced cardiovascular deaths by 21% compared to Plavix in a big clinical trial. That would mean that if everyone eligible for Brilinta got it, 100,000 lives would be saved.

But the reality is that doctors have been skeptical of that data because in the part of that trial that was run in North America, the benefit was less clear. AstraZeneca says that this may have been due to an interaction of Brilinta and aspirin and that, according to current cardiovascular guidelines, doctors should be prescribing less aspirin anyway.

Another key data point Astra presented was that blood levels of troponin, a muscle protein released by the heart during a heart attack, predict which patients get the most benefit from Brilinta. This data is not in AstraZeneca’s label, but a spokeswoman said that she believed it would be something the company can market to doctors.

A lot of what Astra will do in the short term on Brilinta will be blocking and tackling. It needs to pay bigger rebates to insurers to make sure that patients can get cheap access to the drug. (This is how discounts happen in the American insurance system: the patient pays a co-payment and the insurer pays full price for the drug, but then the drug maker gives the insurer money back to make the end cost cheaper.) It will also be doing a lot of medical marketing, involving its internal experts or paid, external doctors, to get the word out about the benefits of Brilinta.

Brilinta has other advantages (it stops acting quickly) and disadvantages (it must be given twice a day). But the other big question for expanding results is whether large clinical trials that are now ongoing will show that it works in a broader array of heart patients. Astra is starting a big trial to show Brilinta prevents strokes. These trials are risky and expensive, but there will be a big payoff if they work.

Astra has some other commercial levers to point to. It’s diabetes pill Onglyza, which is sold with Bristol-Myers Squibb, will have results in a big study of its efficacy in preventing heart disease before a similar study of Merck’s top-selling Januvia, which started first. Soriot has smart ideas about which drugs to advance into later testing. But Brilinta is going to be the biggest single indicator of whether Soriot’s new strategies are paying off.

SOURCE:

http://www.forbes.com/sites/matthewherper/2013/03/21/can-pascal-soriot-turn-around-astrazeneca-it-may-come-down-to-one-drug/

BRILINTA is an antiplatelet medication

Taking BRILINTA is a first step in the treatment your physician has chosen for you. At BRILINTA.com, you will find helpful information and useful learning tools to help you complete your course of BRILINTA therapy. Make sure you and your loved ones read through all of the sections.

What is BRILINTA?

BRILINTA is a type of prescription antiplatelet medication for people who have had a recent heart attack or severe chest pain that happened because their heart wasn’t getting enough oxygen and who are being treated with medicines or procedures to open blocked arteries in the heart. BRILINTA is used with aspirin to stop platelets from sticking together and forming a blood clot that could block blood flow to the heart and cause another, possibly fatal, heart attack. Platelets are small cells in the blood that help with normal blood clotting.

Take BRILINTA and aspirin exactly as instructed by your doctor: BRILINTA twice a day, plus one 81-mg aspirin tablet once a day. You should not take a dose of aspirin higher than 100 mg each day because it can affect how well BRILINTA works. Tell your doctor about any medicines you are taking that contain aspirin. Do not take any new medicines that contain aspirin.

Why BRILINTA?

BRILINTA used with aspirin lowers your chance of having another serious problem with your heart or blood vessels such as heart attack, stroke, or blood clots in your stent if you received one. These can be fatal. In fact, in a large clinical study BRILINTA was even better than Plavix® (clopidogrel bisulfate) tablets at lowering your chances of having another heart attack.

BRILINTA is used to lower your chance of having another heart attack or dying from a heart attack, but BRILINTA (and similar drugs) can cause bleeding that can be serious and sometimes lead to death.

Complete the
Course
 Program

IMPORTANT SAFETY INFORMATION ABOUT BRILINTA

BRILINTA is used to lower your chance of having another heart attack or dying from a heart attack or stroke, but BRILINTA (and similar drugs) can cause bleeding that can be serious and sometimes lead to death. Instances of serious bleeding, such as internal bleeding, may require blood transfusions or surgery. While you take BRILINTA, you may bruise and bleed more easily and be more likely to have nosebleeds. Bleeding will also take longer than usual to stop.

Call your doctor right away if you have any signs or symptoms of bleeding while taking BRILINTA, including: severe, uncontrollable bleeding; pink, red, or brown urine; vomit that is bloody or looks like coffee grounds; red or black stool; or if you cough up blood or blood clots.

Do not stop taking BRILINTA without talking to the doctor who prescribes it for you. People who are treated with a stent, and stop taking BRILINTA too soon, have a higher risk of getting a blood clot in the stent, having a heart attack, or dying. If you stop BRILINTA because of bleeding, or for other reasons, your risk of a heart attack or stroke may increase. Tell all your doctors and dentists that you are taking BRILINTA. To decrease your risk of bleeding, your doctor may instruct you to stop taking BRILINTA 5 days before you have elective surgery. Your doctor should tell you when to start taking BRILINTA again, as soon as possible after surgery.

Take BRILINTA and aspirin exactly as instructed by your doctor. You should not take a dose of aspirin higher than 100 mg daily because it can affect how well BRILINTA works. Tell your doctor if you take other medicines that contain aspirin. Do not take new medicines that contain aspirin.

Do not take BRILINTA if you are bleeding now, especially from your stomach or intestine (ulcer), have a history of bleeding in the brain, or have severe liver problems.

BRILINTA can cause serious side effects, including bleeding and shortness of breath. Call your doctor if you have new or unexpected shortness of breath or any side effect that bothers you or that does not go away. Your doctor can decide what treatment is needed.

Tell your doctor about all the medicines you take, including prescription and nonprescription medicines, vitamins, and herbal supplements. BRILINTA may affect the way other medicines work, and other medicines may affect how BRILINTA works.

Approved uses
BRILINTA is a prescription medicine for people who have had a recent heart attack or severe chest pain that happened because their heart wasn’t getting enough oxygen and who are being treated with medicines or procedures to open blocked arteries in the heart.

BRILINTA is used with aspirin to lower your chance of having another serious problem with your heart or blood vessels such as heart attack, stroke, or blood clots in your stent if you received one. These can be fatal.

Please read Prescribing Information, including Boxed WARNINGS.

Please read Medication Guide.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

If you are without prescription coverage and cannot afford your medication, AstraZeneca may be able to help. For more information, please visit www.AstraZeneca.com.

This product information is intended for US consumers only.

BRILINTA is a trademark of the AstraZeneca group of companies.

Plavix® is a registered trademark of sanofi-aventis.

©2012 AstraZeneca.706809-1789005 8/12

SOURCE:

http://www.brilinta.com/antiplatelet-prescription-medication.aspx#au

http://www1.astrazeneca-us.com/pi/brilinta.pdf

BRILINTA (ticagrelor)

Ticagrelor (trade name Brilinta in the US, Brilique and Possia in the EU) is a platelet aggregation inhibitor produced by AstraZeneca. The drug was approved for use in the European Union by the European Commission on December 3, 2010.[1][2] The drug was approved by the US Food and Drug Administrationon July 20, 2011.[3]

Indications

Ticagrelor is indicated for the prevention of thrombotic events (for example stroke or heart attack) in patients with acute coronary syndrome or myocardial infarction with ST elevation. The drug is combined with acetylsalicylic acid unless the latter is contraindicated.[4] Treatment of acute coronary syndrome with ticagrelor as compared with clopidogrel significantly reduces the rate of death.[5]

Contraindications

Contraindications for ticagrelor are: active pathological bleeding and a history of intracranial bleeding, as well as reduced liver function and combination with drugs that strongly influence activity of the liver enzymeCYP3A4, because the drug is metabolized via CYP3A4 and excreted via the liver.[4]

Adverse effects

The most common side effects are shortness of breath (dyspnea, 14%)[6] and various types of bleeding, such as hematomanosebleedgastrointestinalsubcutaneous or dermal bleeding. Allergic skin reactions such as rash and itching have been observed in less than 1% of patients.[4]

Physical and chemical properties

Ticagrelor is a nucleoside analogue: the cyclopentane ring is similar to the sugar ribose, and the nitrogen rich aromatic ring system resembles the nucleobase purine, giving the molecule an overall similarity toadenosine. The substance has low solubility and low permeability under the Biopharmaceutics Classification System.[1]

Ticagrelor as a nucleoside analogue

The nucleoside adenosinefor comparison

Pharmacokinetics

Ticagrelor is absorbed quickly from the gut, the bioavailability being 36%, and reaches its peak concentration after about 1.5 hours. The main metabolite, AR-C124910XX, is formed quickly via CYP3A4 by de-hydroxyethylation at position 5 of the cyclopentane ring.[7] It peaks after about 2.5 hours. Both ticagrelor and AR-C124910XX are bound to plasma proteins (>99.7%), and both are pharmacologically active. Blood plasma concentrations are linearly dependent on the dose up to 1260 mg (the sevenfold daily dose). The metabolite reaches 30–40% of ticagrelor’s plasma concentrations. Drug and metabolite are mainly excreted via bile and feces.

Plasma concentrations of ticagrelor are slightly increased (12–23%) in elderly patients, women, patients of Asian ethnicity, and patients with mild hepatic impairment. They are decreased in patients that described themselves as ‘coloured’ and such with severe renal impairment. These differences are considered clinically irrelevant. In Japanese people, concentrations are 40% higher than in Caucasians, or 20% after body weight correction. The drug has not been tested in patients with severe hepatic impairment.[4]

Mechanism of action

Like the thienopyridines prasugrelclopidogrel and ticlopidine, ticagrelor blocks adenosine diphosphate (ADP) receptors of subtype P2Y12. In contrast to the other antiplatelet drugs, ticagrelor has a binding site different from ADP, making it an allosteric antagonist, and the blockage is reversible.[8] Moreover, the drug does not need hepatic activation, which might work better for patients with genetic variants regarding the enzyme CYP2C19 (although it is not certain whether clopidogrel is significantly influenced by such variants).[9][10][11]

Comparison with clopidogrel

The PLATO trial, funded by AstraZeneca, in mid-2009 found that ticagrelor had better mortality rates than clopidogrel (9.8% vs. 11.7%, p<0.001) in treating patients with acute coronary syndrome. Patients given ticagrelor were less likely to die from vascular causes, heart attack, or stroke but had greater chances of non-lethal bleeding (16.1% vs. 14.6%, p=0.0084), higher rate of major bleeding not related to coronary-artery bypass grafting (4.5% vs. 3.8%, P=0.03), including more instances of fatal intracranial bleeding. Rates of major bleeding were not different. Discontinuation of the study drug due to adverse events occurred more frequently with ticagrelor than with clopidogrel (in 7.4% of patients vs. 6.0%, P<0.001)[5] The PLATO trial showed a statistically insignificant trend toward worse outcomes with ticagrelor versus clopidogrel among US patients in the study – who comprised 1800 of the total 18,624 patients. The HR actually reversed for the composite end point cardiovascular (death, MI, or stroke): 12.6% for patients given ticagrelor and 10.1% for patients given clopidogrel (HR = 1.27). Some believe the results could be due to differences in aspirin maintenance doses, which are higher in the United States.[12] Others state that the central adjudicating committees found an extra 45 MIs in the clopidogrel (comparator) arm but none in the ticagrelor arm, which improved the MI outcomes with ticagrelor. Without this adjudication the trials’ primary efficacy outcomes should not be significant[13]

Consistently with its reversible mode of action, ticagrelor is known to act faster and shorter than clopidogrel.[14] This means it has to be taken twice instead of once a day which is a disadvantage in respect of compliance, but its effects are more quickly reversible which can be useful before surgery or if side effects occur.[4][15]

Interactions

Inhibitors of the liver enzyme CYP3A4, such as ketoconazole and possibly grapefruit juice, increase blood plasma levels and consequently can lead to bleeding and other adverse effects. Conversely, drugs that are metabolized by CYP3A4, for example simvastatin, show increased plasma levels and more side effects if combined with ticagrelor. CYP3A4 inductors, for example rifampicin and possibly St. John’s wort, can reduce the effectiveness of ticagrelor. There is no evidence for interactions via CYP2C9.

The drug also inhibits P-glycoprotein (P-gp), leading to increased plasma levels of digoxinciclosporin and other P-gp substrates. Ticagrelor and AR-C124910XX levels are not significantly influenced by P-gp inhibitors.[4]

In the US a boxed warning states that use of ticagrelor with aspirin doses exceeding 100 mg/day decreases the effectiveness of the medication.[16]

References

  1. a b “Assessment Report for Brilique”European Medicines Agency. January 2011.
  2. ^ European Public Assessment Report Possia
  3. ^ “FDA approves blood-thinning drug Brilinta to treat acute coronary syndromes”. FDA. 20 July 2011.
  4. a b c d e f Haberfeld, H, ed. (2010) (in German). Austria-Codex (2010/2011 ed.). Vienna: Österreichischer Apothekerverlag.
  5. a b Wallentin, Lars; Becker, RC; Budaj, A; Cannon, CP; Emanuelsson, H; Held, C; Horrow, J; Husted, S et al. (August 30, 2009). “Ticagrelor versus Clopidogrel in Patients with Acute Coronary Syndromes”NEJM 361 (11): 1045–57. doi:10.1056/NEJMoa0904327PMID 19717846.
  6. ^ Brilinta: Highlights of prescribing information
  7. ^ Teng, R; Oliver, S; Hayes, MA; Butler, K (2010). “Absorption, distribution, metabolism, and excretion of ticagrelor in healthy subjects”. Drug metabolism and disposition: the biological fate of chemicals 38 (9): 1514–21. doi:10.1124/dmd.110.032250PMID 20551239.
  8. ^ Birkeland, Kade; Parra, David; Rosenstein, Robert (2010). “Antiplatelet therapy in acute coronary syndromes: focus on ticagrelor”Journal of Blood Medicine 1: 197–219.
  9. ^ H. Spreitzer (February 4, 2008). “Neue Wirkstoffe – AZD6140” (in German). Österreichische Apothekerzeitung (3/2008): 135.
  10. ^ Owen, RT, Serradell, N, Bolos, J (2007). “AZD6140”. Drugs of the Future 32 (10): 845–853. doi:10.1358/dof.2007.032.10.1133832.
  11. ^ Tantry, Udaya S; Bliden, Kevin P (2010). “First Analysis of the Relation Between CYP2C19 Genotype and Pharmacodynamics in Patients Treated With Ticagrelor Versus Clopidogrel”. Circulation: Cardiovascular Genetics 3: 556–566. doi:10.1161/CIRCGENETICS.110.958561.
  12. ^ Bernardo Lombo, José G Díez. Ticagrelor: the evidence for its clinical potential as an oral antiplatelet treatment for the reduction of major adverse cardiac events in patients with acute coronary syndromes Core Evid. 2011; 6: 31–42. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3065559/
  13. ^ Serebruany VL, Atar D. Viewpoint: Central adjudication of myocardial infarction in outcome-driven clinical trials—Common patterns in TRITON, RECORD, and PLATO? Thromb Haemost 2012; DOI: 10.1160/TH12-04-0251. http://www.theheart.org/article/1433145/print.do
  14. ^ Miller, R (24 February 2010). “Is there too much excitement for ticagrelor?”. TheHeart.org.
  15. ^ H. Spreitzer (17 January 2011). “Neue Wirkstoffe – Elinogrel” (in German). Österreichische Apothekerzeitung (2/2011): 10.
  16. ^ July 20, 2011 AstraZeneca: Ticagrelor (Brilinta) Gains FDA Approval Larry Husten cardiobrief.org/2011/07/20/astrazeneca-ticagrelor-brilinta-gains-fda-approval/

SOURCE:

 http://en.wikipedia.org/wiki/Ticagrelor

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English: Amino acid sequence of the molecule o...

English: Amino acid sequence of the molecule of the brain natriuretic peptide (BNP) 32 (functional). Português: Sequência de aminoácidos da molécula de BNP 32 (funcional). (Photo credit: Wikipedia)

Assessing Cardiovascular Disease with Biomarker

Author and Curator: Larry H Bernstein, MD, FCAP

 

A Changing expectation from cardiac biomarkers.

This article on Assessing Cardiovascular Disease with Biomarkers will demonstrate the unique role in the discipline evolution that each of the following biomarkers has played in our understanding of CVD risk:

The article is introduced with an entire section on the evolution of our knowledge of cardiac biomarkers and how concepts from thermodynamics have transformed
the way we investigate biochemical mechanisms, and how we have gone from a macro- to a micro- landscape of high complexity.  The same concepts from physics
have also transformed the mathematical stage upon which we model data.  BIG Data is not just about business!  We have entered a new domain of knowledge enabling.

(1)  Enzymes and Isoenzymes

  • AST, ALT, LD, alkaline phosphatase
  • Isoenzymes evolution and genomic loci for polypeptides
  • Emergence of pathway divergence and regulation from gene-loci peptide changes
  • A reflection to implications for biomarkers and therapeutic development based on critical links

(2)  Natriuretic Peptides

  •       Cause of Death: silent cardiac target organ damage (cTOD) (no so sign of cardiac disease)
  •       B Type natriuretic peptide in evolution of CHF
  •       2D and Doppler echocardiography and BNP serum level
  •       Amino terminal pro B-type Natriuretic Peptide
  •       Renal Effect on NT-proBNP
  •       pro-atrial natriuretic peptide

(3)     CRP as Biomarker, theory that lowering the C-reactive protein (CRP) level with statin therapy is predictive of cardiovascular outcomes independent of lowering the low-density lipoprotein (LDL) cholesterol level

(4)     CRP as an Inflammatory Agent

Acute phase reaction is a systemic response: physiological condition  in the beginning of an inflammatory process.

(5)     troponins and hs-troponins (I, T)

(6)     New Candidate  Biomarkers for NSTEMI

(7)    Guidelines for Cardiovascular Risk Assessment

(8)     Statistical Issues to be Resolved

Historical perspective

The use of cardiac markers emerged in the late 1950s, when the physician was faced with the problem of a patient with recent onset of squeezing, crushing, or heaviness in the chest, with or without a Q-wave or definitive ST elevation (acute injury), and perhaps a non specific elevation of the neutrophil count.   A medical student at Albert Einstein Medical school at the time, Arthur Karmen identified the first enzymatic test for acute myocardial infarct (MI), serum glutamic oxaloacetic acid transaminase (SGOT), which is renamed Aspartate Aminotransferase (AST) in a seminal study with Wroblewski and LaDue[1].  The enzyme is ubiquitous, and the authors published another observation that the SGPT, now referred to as Alanine Aminotransferase, has a greater specific activity in liver and myocardial infarct can be distinguished from necrotizing liver disease by using AST and ALT.  These two enzymes were among the three enzymes,with lactate dehydrogenase (LD) and alkaline phosphatase that appeared on the original Technicon (later Siemens) SMA-12 profile, prior to the designated panels used today.  At that time it was common for the pathologist to stain the heart lesion at autopsy in identifying the “ischemic necrosis” postmortem.

In 1957 Hunter and Markert described the five isoenzymes of lactate dehydrogenase, the most anodal migrating pattern was associated with heart and the most cathodal isoenzymes with liver, the five bands being combinations of two subunits.  These were described as different variants of the same enzyme having identical functions, but different tissue specific patterns, such that,  enzyme variants have altered gene loci that results in an amino acid change but catalyze the same reaction.  When mutation modifies the enzymatic catalysis, or its pattern of gene expression, then any of two (or more) variants may be favoured by natural selection and become specialized to different cell environments.  His group suggested that a single gene might somehow encode an array of isozymes differing in “structural variations,” a concept that seems to presage our current understanding of alternative mRNA splicing and post-translational protein modification. A former student of George beadle, he transformed the “concept of one gene one enzyme”  to “one gene one polypeptide”. By treating the enzyme with denaturing agents it was learned that LDH is a tetramer of two types of polypeptide chains (Appella and Markert, 1961). Thus the multiple-gene hypothesis was partially correct: Two different LDH subunits, each encoded by a distinct gene, re-sort themselves in various tetrameric combinations to give rise to five different isozymes (Markert, 1963). During the succeeding years Markert and his students and postdocs elucidated how the study of isozymes could contribute to our understanding of the biochemical variation that underlies cell differentiation and evolution, culminating in the new perspective presented in a Science paper (Markert et al., 1975) entitled “Evolution of a Gene.”

In the early 1960’s Nathan Kaplan postulated that the major LD-isoenzyme types were associated with fundamental differences in the metabolism of the tissue of origin, either catabolic (heart) or anabolic (liver), and skeletal muscle would appear to be in the same class as liver (ignore the ratio of fast and slow twitch), which was elaborated on further by studies of the flight wing patterns of birds.   These isoenzymes not only had different migration in an electrophoretic field and could be separated chromatographically, but they also had different kinetic properties. They all have the same Km, but the purified heart LD is inhibited by a ternary complex of the enzyme, the NAD, and pyruvate that forms, slowing the reaction in the forward direction (pyruvate to lactate).

At about the same time, Masahiro Chiga discovered that adenylate kinase, the enzyme that converts ATP to ADP, from skeletal muscle can be inhibited by inorganic S (myokinase), which led Bernstein and Russell to publish on the identification of adenylate kinase from heart in myocardial infarction using sulfhydryl inhibition in J Molec Cellular Cardiology.  Burton Sobel in the early 1970s showed that CK and the MB isoenzyme of CK, which has a more rapid increase and disappearance than the AST or LD ,  could be used to estimate the amount of cardiac damage in MI.   This meant that a test could be done at any time of day or night with a result in less than an hour.  He applied this to determining whether the extent of infarction was an important determinant of prognosis after myocardial infarction and furthermore, whether the extent of infarction could be modified by interventions that reduce myocardial oxygen requirements or increase myocardial oxygen supply. This work has had a major impact on how patients with acute myocardial infarction are treated and led to a reduction of mortality secondary to treatments, such as thrombolysis, that were validated initially with the methods developed. This led to an immunoassay for CK isoenzyme MB that was offered by Roche on the Cobas analyzer, and by Dupont on the ‘aca’. What emerged is a new imperative to reduce infarct size under the rubrick – “Time is Muscle”.

References

  1. Karmen Arthur, Wróblewski Felix, LaDue John S. TRANSAMINASE ACTIVITY IN HUMAN BLOOD. J Clin Invest. 1955; 34(1):126–133.
  2. LADUE JS, WROBLEWSKI F, KARMEN A. Serum glutamic oxaloacetic transaminase activity in human acute transmural myocardial infarction. Science 1956; 75(11).
  3. Hunter, R. L. and C.L. Merkert. (1957) Histochemical demonstration of enzymes separated by zone electrophoresis in starch gels. Science 125: 1294-1295.
  4. Bernstein L, Kerrigan M, Maisel H. Lactic dehydrogenase isoenzymes in lens and cornea. Exp Eye Res 1965; 5(3):999-1005. ICID: 844979
  5. Nathan O. Kaplan Papers. MSS 0099. UC San Diego::Mandeville Special Collections Library.

Enzyme-coenzyme-substrate complex. of pyridine nucleotide depend. dehydrogenases 1958.  box 39, folder 5.
Enzymatic studies with analogues of diphosphopyridine nucleotide 1959. box 39, folder 12.
Heterogeneity of the lactic dehydrogenases of new-born and adult rat heart as determined with enzyme analogs 1961. box 39, folder 37.
Regulatory effects of enzyme action 1961. box 39, folder 38.
Inhibition of dehydrogenase reactions by a substance formed from reduced dpn 1961. box 39, folder 40.
Lactic dehydrogenases: functions of the two types 1964. box 39, folder 67.
Lactate dehydrogenase – structure and function. 1964. box 40, folder 4.
Role of the two types of lactic dehydrogenases 1964. Box 40, folder 9.
Structural and functional properties of h and m subunits of lactic dehydrogenase 1965. Box 40, folder 12.

  • Bernstein LH, Everse J, Shioura N, Russell PJ. Detection of cardiac damage using a steady state assay for lactate dehydrogenase isoenzymes in serum. J Mol Cell Cardiol 1974; 6(4):297-315. ICID: 825597
  • Bernstein LH, Everse J.  Determination of the isoenzyme levels of lactate dehydrogenase. Methods Enzymol 1975; 41 47-52.
  • Bernstein LH. Automated kinetic determination of lactate dehydrogenase isoenzymes in serum. Clin Chem 1977; 23(10):1928-1930. ICID: 825616
  • Bernstein LH, Scinto P. Two methods compared for measuring LD-1/total LD activity in serum. Clin Chem 1986; 32(5):792-796. ICID: 825581
  1. Shell WE, Kjekshus JK, Sobel BE: Quantitative assessment of the extent of myocardial infarction in the conscious dog by means of analysis of serial changes in serum creatine phosphokinase activity. J Clin Invest 50:2614-2626, 1971.
  2. Bergmann SR, Fox KAA, Ter-Pogossian MM, Sobel BE (Washington University), Collen D (University of Leuven): Clot-selective coronary thrombolysis with tissue-type plasminogen activator. Science 220:1181-1183, 1983.
  3. Van de Werf F, Ludbrook PA, Bergmann SR, Tiefenbrunn AJ, Fox KAA, de Geest H, Verstraete M, Collen D, Sobel BE: Coronary thrombolysis with tissue-type plasminogen activator in patients with evolving myocardial infarction. N Engl J Med 310:609-613, 1984.
  • Adan J, Bernstein LH, Babb J. Can peak CK-MB segregate patients with acute myocardial infarction into different outcome classes?  Clin Chem 1985; 31(2):996-997. ICID: 844986
  • Bernstein LH, Reynoso G.  Creatine kinase B-subunit activity in serum in cases of suspected myocardial infarction: a prediction model based on the slope of MB increase and percentage CK-MB activity. Clin Chem 1983; 29(3):590-592. ICID: 825549
  • Bernstein LH, Horenstein JM, Sybers HB, Russell PJ.  Adenylate kinase in human tissue. II. Serum adenylate kinase and myocardial infarction. J Mol Cell Cardiol 1973; 5(1):71-85. ICID: 825590

A Metabolic Functional Meaning of Existence of Isoenzymes

There are many examples of  isozymes, such as glucokinase, a variant of hexokinase which is not inhibited by glucose 6-phosphate. It has different regulatory features and lower affinity for glucose (compared to other hexokinases). Alkaline and acid phosphatase isoenzymes were used briefly for a time in clinical diagnostics.  These isoenzymes are oligomeric proteins that have distinct subunits that affect their binding with substrate.  A distinctive type of protein that can form two or more different homo-oligomers, comes apart and changes shape to convert between forms is called a morpheein. The alternate shape may reassemble to a different oligomer, and the shape of the subunit dictates which oligomer is formed. Morpheeins can interconvert between forms under physiological conditions and can exist as an equilibrium of different oligomers. Features of morpheeins can be exploited for drug discovery. A small molecule compound can shift the equilibrium either by blocking or favoring formation of one of the oligomers. The equilibrium can be shifted using a small molecule that has a preferential binding affinity for only one of the alternate morpheein forms. This introduces the concept of allostericity.  Most allosteric effects can be explained by a model put forth by Monod, Wyman, and Changeux, and also by a model described by Koshland, Nemethy, and Filmer. Both postulate that enzyme subunits exist in one of two conformations, tensed (T) or relaxed (R), and that relaxed subunits bind substrate more readily than those in the tense state.  This concept provides a foundation for another generation of biomarkers than was the focus of the 20th century, and only has been investigated since the 1980’s, and takes another dimension after the completion of the Human Genome Project, opening a “Pandora’s box”. This moved biomedical science forward into an emerging field of ‘OMICs’, which tied small molecules into regulatory processes, transcription, and the possibility of identifying new biomarkers and developing new biomolecules that could modify disease progression.

References

  1. Bu Z, Callaway DJ. “Proteins MOVE! Protein dynamics and long-range allostery in cell signaling”. Adv in Protein Chemistry and Structural Biology 2011; 83: 163–221. doi:10.1016/B978-0-12-381262-9.00005-7. PMID 21570668.
  2.  Monod J, Wyman J, Changeux JP. On the nature of allosteric transitions:A plausible model. J Mol Biol, May 1965; 12:88-118.
  3.   Koshland DE, Némethy G, Filmer D. Comparison of experimental binding data and theoretical models in proteins containing subunits. Biochemistry. Jan 1966; 5(1):365-8
  4.  Jaffe EK. “Morpheeins – a new structural paradigm for allosteric regulation”. Trends Biochem Sci 2005; 30(9): 490–497. doi:10.1016/j.tibs.2005.07.003. PMID 16023348.
  5.  Huang Z, Zhu L, Cao Y, Wu G, Liu X, et al.  ASD: a comprehensive database of allosteric proteins and modulators. Nucleic Acids Res 2011; 39: D663-669

Fundamental Transformative Concepts Carried Over from Physics to Biomolecular Processes.

A colleague once noted that we are learning more and more about less and less.  This is the remarkable evolution of our thinking from macrostates to microstates and segmentation of processes, further leading us to exploration of interactions between states.  This has required a breakdown and a repeated remodeling or resynthesis of ideas based on new findings in science.  It has gradually driven medicial science to a greater dependence on chemistry and physics in underlying principle.  We can better envision the mechanism of evolution from the concepts put forth.

In 1824 Sadi Carnot published the concept that heat is lost in the conversion into work, using the term “caloric”, equivalent to entropy in the second law of thermodynamics.  Clausius then develops the concepts of interior work in 1854, i.e. that “which the atoms of the body exert upon each other”, and exterior work, i.e. that “which arise from foreign influences [to] which the body may be exposed”, anticipating the concept of entropy. He enunciated the passage of the quantity of heat Q from the temperature T1 to the temperature T2 has the equivalence-value entropy, symbolized by S :  dS = Q (1/T2 – 1/T1), which led to his 1865 statement on irreversible heat loss: I propose to name the quantity S the entropy of the system, after the Greek word [τροπη trope], the transformation. I have deliberately chosen the word entropy to be as similar as possible to the word energy.”  In 1876, physicist J. Willard Gibbs, building on the work of Clausius, Hermann von Helmholtz and others, proposed that the measurement of “available energy” ΔG in a thermodynamic system could be mathematically accounted for by subtracting the “energy loss” TΔS from total energy change of the system ΔH, and in 1877, Ludwig Boltzmann formulated the alternative definition of entropy S defined as:

S = kBlnΩ

where

kB is Boltzmann’s constant and

Ω is the number of microstates consistent with the given macrostate.

An analog to thermodynamic entropy is information entropy. Claude Shannon set out to mathematically quantify the statistical nature of “lost information” in phone-line signals  and developed  a concept of information entropy, a fundamental cornerstone of information theory. The close similarity between his new quantity and earlier work in thermodynamics is attributed to a visit and discussion with Jon von Neumann in 1949. Shannon then called the “measure of uncertainty” or attenuation in phone-line signals with reference to his new information theory.  This led to the elucidation of a signal (as opposed to noise, by Solomon Kullback, which became the basis for the measure of an optimum diagnostic decision point of a laboratory test by Bernstein and Rudolph, related to Eugene Rypka’s “Syndromic Clustering”.  The loop was closed by the Japanese mathematician Akaike, who brought Fisher’s statistical formulations and Kullback-Liebler distance into alignment.   This is not a digression because it has been central to underlying principles in resolution in spectroscopy, and to classification of biochemical molecular features.

Although Boltzmann first linked entropy and probability in 1877, it seems the relation was never expressed with a specific constant until Max Planck first introduced k, and gave an accurate value for it (1.346×10−23 J/K, about 2.5% lower than today’s figure), in his derivation of the law of black body radiation in 1900–1901. Before 1900, equations involving Boltzmann factors were not written using the energies per molecule and the Boltzmann constant, but rather using a form of the gas constant R, and macroscopic energies for macroscopic quantities of the substance. The iconic terse form of the equation S = k log W on Boltzmann’s tombstone is in fact due to Planck, not Boltzmann. Planck actually introduced it in the same work as his h. Planck noted in his 1920 Nobel Prize acceptance : “:This constant is often referred to as Boltzmann’s constant, although, to my knowledge, Boltzmann himself never introduced it — a peculiar state of affairs.”  The Kullback–Leibler divergence (also information divergence, information gain, relative entropy, or KLIC) is a non-symmetric measure of the difference between two probability distributions P and Q, was  introduced by Solomon Kullback and Richard Leibler in 1951. KL-divergence of a model from reality may be estimated, to within a constant additive term, by a function (like the squares summed) of the deviations observed between data and the model’s predictions. When trying to fit parametrized models to data there are various estimators which attempt to minimize Kullback–Leibler divergence, such as, the familiar maximum likelihood  estimator.

References

  1. Planck, Max (2 June 1920), The Genesis and Present State of Development of the Quantum Theory (Nobel Lecture)
  2. Kalinin M, Kononogov S. “Boltzmann’s Constant, the Energy Meaning of Temperature, and Thermodynamic Irreversibility”, Measurement Techniques 2005; 48 (7): 632–36, doi:10.1007/s11018-005-0195-9
  3. Kullback S, Leibler RA “On Information and Sufficiency”. Annals of Mathematical Statistics 1951; 22 (1): 79–86. doi:10.1214/aoms/1177729694. MR 39968.
  4. Kullback S (1959) Information theory and statistics (John Wiley and Sons, NY).
  5. Jaynes ET(1957) Information theory and statistical mechanics, Physical Review 106:620
  6. Jaynes ET(1957) Information theory and statistical mechanics II, Physical Review 108:171
  7. Burnham KP and Anderson DR. (2002) Model Selection and Multimodel Inference: A Practical Information-Theoretic Approach, Second Edition (Springer Science, New York) ISBN 978-0-387-95364-9.
  8. Rudolph RA, Bernstein LH, Babb J.  Information induction for predicting acute myocardial infarction. Clin Chem 1988; 34(10):2031-2038. ICID: 825568

A New Imperative

Cardiovascular Biomarkers

I. BNP:

[A] Aids in the Prevention of Cardiac Events by Detecting Silent Ischemic Lesions and Selecting Patients for Imaging

12/17/12 · Emily Humphreys

Physicians use risk factors, such as history, exercise level, diabetes, blood pressure, lipid profiles, and other laboratory measurements to ascertain risk for cardiac events, which are not foolproof in predicting all cardiac events. Nonetheless, 40% to 50% of sudden cardiac deaths (SCD) occur before risk factors are able to predict cardiac events.2,3 Those who die suddenly with no so sign of cardiac disease often have silent cardiac target organ damage (cTOD).  While patients with silent ischemia have a 21-fold increase in risk of a coronary event.4 It has also been shown that cTODs such as left ventricular hypertrophy (LVH), left ventricular systolic dysfunction (LVSD), left ventricular diastolic dysfunction (LVDD), and left atrial enlargement (LAE) each independently predict cardiovascular events5,6,7,8 Nadir et al. hypothesized that identification of silent cTOD would aid in the prevention of cardiovascular events, including SCDs.9 To identify cTOD present, The Nadir group evaluated several known cardiac biomarkers including: B-type natriuretic peptide (BNP), high-sensitivity cardiac troponin T (hs-cTnT), microalbuminuria, the estimated glomerular filtration rate, and uric acid.  The lab results of 300 asymptomatic individuals recruited for the study were compared with primary screening using transthoracic echocardiography, stress echocardiography, and/or myocardial perfusion imaging.
  • 34% of study volunteers had evidence of a cTOD. Out of all biomarkers analyzed, BNP levels were significantly higher in those with cTOD compared with those without. BNP levels were also higher in those who had more than one form of cTOD compared with those who had a single form of cTOD.
  • Hs-cTnT also performed well, but BNP levels had the highest correlation to imaging data. The gold standard diagnostic tool for cardiovascular disease is imaging studies, such as echocardiography.
  • It is not standard practice to investigate healthy individuals for possible cTOD and would be costly and time consuming to perform imaging on these individuals.
  • Biomarkers like BNP could be used as a primary screening tool with follow-up image studies performed, if necessary.

The eventual hope is to reduce the mortality of cardiovascular diseases and prevent silent cTOD from leading to more serious and potentially life-threatening cardiac events.

References

1. Roger, V.L. (2012) ‘AHA statistical update: Heart disease and stroke statistics-2012 update. A report from the american heart association‘, Circulation, 125 (2012), (pp. e2-e220)

2. Chiuve, S.E., et al., (2006) ‘Healthy lifestyle factors in the primary prevention of coronary heart disease among men: Benefits among users and nonusers of lipid-lowering and antihypertensive medications‘ Circulation, 114 (2006), (pp. 160-167)

3.De Vreede-Swagemakers, J.J., et al. (1997) ‘Out-of-hospital cardiac arrest in the 1990s: A population-based study in the Maastricht area on incidence, characteristics and survival‘, Journal of the American College of Cardiology, 30 (1997), (pp. 1500-1505)

4. Rutter, M.K., et al. (2002) ‘Significance of silent ischemia and microalbuminuria in predicting coronary events in asymptomatic patients with type 2 diabetes‘, Journal of the American College of Cardiology, 40 (2002), (pp. 56-61)

5. Tsang, T.S., et al. (2003) Prediction of risk for first age-related cardiovascular events in an elderly population: The incremental value of echocardiography‘, Journal of the American College of Cardiology, 42 (2003), (pp. 1199-1205)

6. Gosse, P., (2005) ‘Left ventricular hypertrophy—the problem and possible solutions‘,The Journal of International Medical Research, 33 (Suppl 1) (2005), (pp. 3A-11A)

7. Benjamin, E.J., et al. (1995) ‘Left atrial size and the risk of stroke and death‘ The Framingham Heart Study Circulation, 92 (4), (pp. 835-41)

8. Redfield, M.M., et al. (2003) ‘Burden of systolic and diastolic ventricular dysfunction in the community: Appreciating the scope of the heart failure epidemic‘, JAMA, 289 (2003), (pp. 194-202)

9. Nadir, M.A., et al., (2012) ‘Improving the primary prevention of cardiovascular events by using biomarkers to identify individuals with silent heart disease‘, Journal of American College of Cardiology, 60 (11), (pp. 960-968) Tags: 

[B] Evaluating CHF patients in the emergency department

The role of B-type natriuretic peptide in the evaluation of congestive heart failure patients in emergency department

Congestive heart failure (CHF) is a severe cardiovascular disorder seen in the Emergency Department (ED). B-type Natriuretic Peptide (BNP) is usually ordered to evaluate the CHF severity.

However, it is difficult to interpret serum BNP level when different clinical entities existed.

The aim of this study is to illustrate the correlation between serum BNP level and

  • relevant clinical variables and
  • further determine the role of serum BNP in different CHF patients.

High variability of serum BNP levels exists in CHF patients with weak-to-moderate correlation effects particularly on obesity and diastolic/systolic HF.

Physicians should be cautious on interpreting BNP in different CHF populations.

[C]   NT-proBNP compared with ECHO

Comparison of N-Terminal Pro B-Natriuretic Peptide and Echocardiographic Indices in Patients with Mitral Regurgitation.  Shokoufeh Hajsadeghi1, Niloufar Samiei2, Masoud Moradi3, Maleki Majid2, et al. Corresponding author email: masoud_moradi65@yahoo.com

Abstract

Introduction: Echocardiographic indices can form the basis of the diagnosis of systolic and diastolic left ventricular (LV) dysfunction in patients with Mitral regurgitation (MR). However, using echocardiography alone may bring us to a diagnostic dead-end. The aim of this study was to compare N-Terminal pro B-natriuretic peptide (BNP) and echocardiographic indices in patients with mitral regurgitation.

Methods: 2D and Doppler echocardiography and BNP serum level were obtained from 54 patients with organic mild, moderate and severe MR.

Results: BNP levels were increased with symptoms in patients with mitral regurgitation (NYHAI: 5.7 ± 1.1, NYHAII: 6.9 ± 1.5, NYHAIII: 8.3 ± 2 pg/ml, P , 0.001). BNP plasma level were significantly correlated with MPI (myocardial performance index)(r = 0.399, P = 0.004), and following echocardiographic indices: LVEDV (r = 0.45, P , 0.001), LVESV (r = 0.54, P , 0.001), LVEDD (r = 0.48, P , 0.001), LVESD (r = 0.54, P , 0.001), dp/dt (r = −0.32, P = 0.019) and SPAP (r = 0.4, P = 0.006).

Conclusion: The present study showed that BNP may be useful in patients with MR and may confirm echocardiographic indices.

Keywords: mitral regurgitation, N-Terminal pro-B natriuretic peptide, echocardiographic indices.

The hypothesis assumes that there is a linear sequence of most effective screening that comes out of this study, from a b-type natriuretic peptide to the imaging.  It’s not clear that that is the case, and moreover, silent myocardial infarct is taken and lumped with other serious conditions affecting the myocardium, presumably through compromise of the end-artery circulation to the heart (R, L, and circumflex coronaries).  There is no mention of whether the patients were screened out for peripheral, carotid, or other associated artery disease (superior mesenteric).

I’ll assume that that is the case.  I see a problem with a linear, monothetic, “gold standard” approach, when the disease and the diagnosis of it is multivariate and requires a method that uses a classificatory approach.  We’ll return to that.

English: A Wiggers diagram, showing the cardia...

English: A Wiggers diagram, showing the cardiac cycle events occuring in the left ventricle. (Photo credit: Wikipedia)

[D]  reference normal for NT-proBNP

ABSTRACT

Background: The natriuretic peptides, B-type natriuretic peptide (BNP) and NT-proBNP that have emerged as tools for diagnosing congestive heart failure (CHF) are affected by age and renal insufficiency (RI).This study evaluates the reference value for interpreting NT-proBNP concentrations. Increasing concentrations of NT-proBNP are associated with co-morbidities, not merely CHF, resulting in altered volume status or myocardial filling pressures.

Methods: NT-proBNP was measured in a population with normal trans-thoracic echocardiograms (TTE) and free of anemia or renal impairment.

Selection of Patients: Study participants were seen in acute care for symptoms of shortness of breath suspicious for CHF.

Results: The median NT-proBNP for patients under 50 years is 27.6 pg/ml with an upper limit of 445 pg/ml, and for patients over 50 years the median was 142.3 pg/ml with an upper limit of 475.3 pg/ml. We introduce a transformed NT-proBNP that normalizes for decrease in glomerular filtration rate and eliminates the age factor.

Conclusion: We suggest that NT-proBNP levels can be more accurately interpreted only after removal of the major co-morbidities that affect an increase in this peptide in serum. The PRIDE study guidelines should be applied until presence or absence of comorbidities is diagnosed. With no comorbidities, the reference range for normal over 50 years of age can be reduced below 800 pg/ml. The effect shown in previous papers likely is due to increasing concurrent comorbidity with age.

Key Words: Congestive Heart Failure, Natriuretic peptides, Anemia, Chronic renal insufficiency

Statistical treatment:

The combined acute and blood donor study sets were kept separate and each analyzed for central tendency, distribution and variability. The two were combined after the comorbidities described above were extracted from the acute care study group. This resulted in a population that should be representative of an unaffected study population that could be used to establish a most representative reference range. The database was replicated several times and then patient rows were randomly deleted until there was an expanded combined and mixed data set with 6,700 entries. All of the database sets were used for analyses.

The results are reported in means with p < 0.05 as the measure of significance for difference between means. Independent Student’s t-tests were applied comparing NT-proBNP and anemia. Univariate ANOVAs were used to compare NT-proBNP levels with varying ranges of hemoglobin and age using SPSS 15.0 (SPSS, Chicago, IL). A linear regression analysis with linear fitting and confidence interval was performed using SYSTAT 12 (SYSTAT, San Jose, CA). The results are reported in means with p < 0.05 as the measure of significance for difference between means. Linear regression analysis, Independent Student’s t and Mann-Whitney tests were applied comparing NT-proBNP for age intervals. Reference range was determined using MedCalc 9.2.0.0 (Mariakerke, Belgium).

We observe the following changes with respect to NT-proBNP and age:
  • Sharp increase in NT-proBNP at over age 50
  • Increase in NT-proBNP at 7 percent per decade over 50
  • Decrease in eGFR at 4 percent per decade over 50
  • Slope of NT-proBNP increase with age is related to proportion of patients with eGFR less than 90
  •  NT-proBNP increase can be delayed or accelerated based on disease comorbidities
Adjustment of the NT-proBNP for eGFR and for age over 50 difference

We have carried out a normalization to adjust for both eGFR and for age over 50:

  • Take Log of NT-proBNP and multiply by 1000
  • Divide the result by eGFR (using MDRD[9] or Cockroft Gault[10])
  • Compare results for age under 50, 50-70, and over 70 years
  • Adjust to age under 50 years by multiplying by 0.66 and 0.56.

GFR (mL/min/1.73 m2) = 186 x (Scr)-1.154 x (Age)-0.203 x (0.742 if female) x (1.210 if African-American) (conventional units)

The equation does not require weight because the results are reported normalized to 1.73 m2 body surface area, which is an accepted average adult surface area.

Comparison of the mean + standard deviation of 607 blood donors and NYMH inpatients for the MDRD and Cockroft Gault (eCG), respectively gave 114.3, 43.7(MDRD); 105.0, 40.1 (eCG). The eCG is predicted by the regression: eCG = 0.059 + 0.918*MDRD. The mean + standard deviation for the age under 50 years and 50 or older is 106.5 + 14.7, 100.9 + 14.5 (MDRD); and 102.5 + 18.5, 98.4 + 20.8 (eCG). These differences are significant at < 0.0001, and 0.010, respectively.

The means comparison of the normalized NT-proBNP (NKLog[NT-proBNP]/eGFR) results in 23.4 and 18.7 for 307 non-donors and 300 donors, significant at p < 0.0001, assuming unequal variance). The difference is not significant for the MDRD normalized NT-proBNP (16.5, 6.6). The normalized by eCG result for 324 under age under 50 years and 283 age 50 years and older is 17.7 versus 18.2, significant at p = 0.0001. The MDRD calculated adjustment is 16.8 vs 16.9, which is not significant. The relationship between these is NKLog(NT-proBNP)/eCG = 4.47 + 0.948*NKLog(NT-proBNP)/MDRD. Figure 4 is a plot of the regression of NKLog(NTproBNP)/MDRD vs NKLog(NTproBNP)/eCG predicted over the full study population.

The reference range for the normalized Klog(NT-proBNP)/MDRD is described by a mean 13.99, median 13.12, and standard deviation 6.14 with a nonparametric upper limit of 24.7. A ROC curve is constructed comparing the NT-proBNP, the NKLog(NTproBNP)/MDRD and the ratio NTproBNP to NKLog(NTproBNP)/MDRD in the expanded full database. The area under the curve is 0.944 (0.938-0.950) for NKLog(NTproBNP)/MDRD with a base of 571 patients with early CHF and 6115 patients without. The reference range for NKLog(proBNP)/MDRD can be referenced to the percentiles as follows: 20, 8.78; 40, 11.92; 60, 14.85; 90, 21.10; 95, 24.73; 97.5, 29.54.

Conclusion: We suggest that NT-proBNP levels can be accurately assessed only after removal of the major confounding co-morbidities that increase this peptide in serum. We established our new range after establishing absence of co-morbidities. The value of this approach for screening purposes is an allowance for a considerably lower reference normal with a higher specificity based on the donor studies and the mixture model. This study finds that the reference range for NT-proBNP is age-dependent past age 50 years, mainly as the change relates to eGFR, and we introduce an age adjusted alternative measure, the normalized NT-proBNP using the MDRD transformation described.

NT-pro BNP reference range with blood donors and patients

Measure                                            NT-proBNP (pg/ml)                         After trimming extremes

Highest                                                    1110                                                                   599.4

Arithmetic mean                                   179.6                                                                   118.2

Geometric mean                                        68.7                                                                      54.4

Median                                                          52.6                                                                     42.6

Standard deviation                                250.5                                                                  150.6

D’Agostino-Pearson                          P = 0.0026                                                    P = 0.0091

97.5%

< 50 years                                                  526.9                                                                 445.0

> 50 years                                                1000                                                                    565.0

Upper Limit of Normal                           772.9                                                                475.3

95% confidence interval                   637.1 – 873.73                                      442.7 – 531.0

Bernstein LH, Zions MY, Alam ME, Haq SA, Heitner JF, et al.  What is the best approximation of reference normal for NT-proBNP? Clinical Levels for Enhanced Assessment of NT-proBNP (CLEAN)

Renal Effect on NT-proBNP

NT-proBNP is excreted by the kidney.  Therefore, GFR has to be taken into account in adjusting the reference range.  BNP, unlike the propeptide, is eliminated 80% by vascular endothelium.  What would be the effect of vascular endothelium erosion?  We don’t know.

The Cockroft Gault equation is widely used in hospitals for adjusting medication doses in hospital patients. The MDRD equation was developed for patients with renal insufficiency and has been shown to be comparable to CG for the population the MDRD is applied. However, the MDRD is only reported to a CLCR of only 60 ml/min and is not validated for age over 65 years. On the other hand, the body weight and BMI, necessary for calculating the CG formula are not routinely done for all patients or in all hospitals. We used 307 inpatients and calculated the MDRD up to 100 ml/min/m2, then used the results to predict the CG. The regression for MDRD versus the CG resulted in an r = 0.884, and a regression equation: CG = -21.1 + 1.172*(MDRD). The initial prediction of CG from MDRDe from 198 of the patients is defined by the regression: CGe = -64.6 + 1.866*MDRDe. (Deming)(95% CI: Intercept -84.5 to -42.8; slope 1.40 to 2.33).  The means, medians, standard deviations, and 97.5th percentiles, respectively, of the age, MDRDe and CGe (calculated from weight data) for the 307 patients are: age- 61.2, 62.0, 17.4, 91.3; MDRD – 121.5, 107.5, 55.9, 212.3; CG – 111.7, 98.7, 51.4, 195.0.

The NT-proBNP was adjusted using a log transform and the estimated GFR (glomerular filtration rate by the original method of Levey et al.  The result for reference corrected Nt proBNP is shown in Table 2.

Table 2.

Kruskal-Wallis test

Data KLOGNTPR
Factor codes MDRD60
Sample size

440

Factor

n

Average Rank

0

344

174.11

1

96

386.73

Test statistic

209.8311

Corrected for ties  Ht

209.8313

Degrees of Freedom (DF)

1

Significance level

P < 0.0001

[E]   Mid-region proANP in Emergency Room

Mid-region pro-hormone markers for diagnosis and prognosis in acute dyspnea: results from the BACH (Biomarkers in Acute Heart Failure) trial.
J Am Coll Cardiol 2010 May 11;55(19):2062-76 (ISSN: 1558-3597)
Maisel A; Mueller C; Nowak R; Peacock WF; Landsberg JW; Ponikowski P; Mockel M; Hogan C; Wu AH; Richards M; Clopton P; Filippatos GS; Di Somma S; Anand I; Ng L; Daniels LB; Neath SX; Christenson R; Potocki M; McCord J; Terracciano G; Kremastinos D; Hartmann O; von Haehling S; Bergmann A; Morgenthaler NG; Anker SD
VA San Diego Healthcare System, San Diego, California 92161, USA. amaisel@ucsd.edu.
OBJECTIVES: Our purpose was to assess the diagnostic utility of midregional pro-atrial natriuretic peptide (MR-proANP) for the diagnosis of acute heart failure (AHF) and the prognostic value of midregional pro-adrenomedullin (MR-proADM) in patients with AHF. BACKGROUND: There are some caveats and limitations to natriuretic peptide testing in the acute dyspneic patient. METHODS: The BACH (Biomarkers in Acute Heart Failure) trial was a prospective, 15-center, international study of 1,641 patients presenting to the emergency department with dyspnea. A noninferiority test of MR-proANP versus B-type natriuretic peptide (BNP) for diagnosis of AHF and a superiority test of MR-proADM versus BNP for 90-day survival were conducted. Other end points were exploratory. RESULTS: MR-proANP (> or =120 pmol/l) proved noninferior to BNP (> or =100 pg/ml) for the diagnosis of AHF (accuracy difference 0.9%). In tests of secondary diagnostic objectives, MR-proANP levels added to the utility of BNP levels in patients with intermediate BNP values and with obesity but not in renal insufficiency, the elderly, or patients with edema. Using cut-off values from receiver-operating characteristic analysis, the accuracy to predict 90-day survival of heart failure patients was 73% (95% confidence interval: 70% to 77%) for MR-proADM and 62% (95% confidence interval: 58% to 66%) for BNP (difference p < 0.001). In adjusted multivariable Cox regression, MR-proADM, but not BNP, carried independent prognostic value (p < 0.001). Results were consistent using NT-proBNP instead of BNP (p < 0.001). None of the biomarkers was able to predict rehospitalization or visits to the emergency department with clinical relevance. CONCLUSIONS: MR-proANP is as useful as BNP for AHF diagnosis in dyspneic patients and may provide additional clinical utility when BNP is difficult to interpret. MR-proADM identifies patients with high 90-day mortality risk and adds prognostic value to BNP. (Biomarkers in Acute Heart Failure [BACH]; NCT00537628). [Copyright 2010 American College of Cardiology Foundation. Published by Elsevier Inc. All rights reserved.].
Comment In: RefSource:J Am Coll Cardiol. 2010 May 11; 55(19):2077-9/PMID:20447529

II. CRP

[A] Predictor of benefit of lowering CRP with statin

Sever PS, Poulter NR, Chang CL, et al; ASCOT Investigators. Evaluation of C-reactive protein prior to and on-treatment as a predictor of benefit from atorvastatin: observations from the Anglo-Scandinavian Cardiac Outcomes Trial. Eur Heart J. 2012;33:486-494

The theory that lowering the C-reactive protein (CRP) level with statin therapy is predictive of cardiovascular outcomes independent of lowering the low-density lipoprotein (LDL) cholesterol level was first advanced by the JUPITER investigators.[1]

  • This study further fueled the debate on whether CRP measurement should be routine for monitoring cardiovascular disease risk. The ASCOT investigators entered this debate when they analyzed data from their trial to determine whether on-statin C-reactive protein level was associated with cardiovascular outcomes.[4]
  • The data in the nested case-control study were from persons recruited to ASCOT in the United Kingdom and Ireland, 9098 of whom were randomly assigned in the blood pressure-lowering arm to receive either amlodipine with or without perindopril or atenolol with or without bendroflumethiazide.[5] Within the whole blood-pressure-lowering group, 4853 persons with a total cholesterol level of 6.5 mmol/L or less (≤ 250 mg/dL) were further randomized to receive atorvastatin or placebo as part of the lipid-lowering arm.[6]
  • For the case-control study, 485 cardiovascular cases were age- and sex-matched with 1367 controls. As expected, the investigators found that baseline LDL cholesterol and CRP levels both predicted cardiovascular events.
  • However, neither the baseline nor the on-treatment CRP level was related to the magnitude of statin efficacy in the prevention of cardiovascular events, whereas the on-treatment LDL cholesterol level was strongly predictive. Including CRP in the Framingham model resulted in a “modest” (2.1%) improvement in risk prediction overall.

The investigators noted that this finding was in line with other prospective studies that showed statistically significant, but modest, absolute improvements with the use of CRP in clinical risk prediction.[7,8] They concluded that “in this hypertensive population selected on the basis of traditional, common coexisting risk factors, CRP did not usefully improve the prediction of cardiovascular events and, critically, reduction in CRP associated with statin therapy was not a predictor of cardiovascular outcome alone or in combination with LDL-cholesterol.”

Eugene Braunwald downplayed the ASCOT investigators’ conclusions in observing “the ASCOT results, albeit quite limited in size, are in fact remarkably similar to those of the [CARE, AFCAPS/TexCAPS, REVERSAL, A to Z], and JUPITER trials, especially in light of the fact that the dose of atorvastatin [in ASCOT] was only 10 mg, while some of the other trials used considerably larger equivalent doses of statins.”

My own take on this is that for at least two decades, there was a belief that the LDL lowering was the main effect of statins, until the (deep frozen) specimens were reexamined from the Framingham study using a hs CRP assay.  The investigation was to determine whether there is a predictor of CVD that is present when the traditional features are not present (which would have to include diabetes and hypertension).  The basis for the use of hsCRP became to identify patients who had sufficient risk to be treated with a statin.  The essential focus seemed to turn on whether statin treatment has efficacy in view of the differential between the LDL or the CRP on the magnitude of the effect.  The muscular effect of a statin then comes into view with the size dose and length of treatment.  However, the CRP measurement identified a relationship between development of the vascular disease and the inflammatory process independently of the STATIN treatment benefit.

Prof. Sever (Medscape): The key result that we found in the initial paper was that CRP, although an independent predictor of cardiovascular events in the hypertensive population, was really only a weak predictor, which is confirmed by the meta-analyses. Furthermore, when you incorporate CRP into a Framingham-style model, it really does not add any benefit or give any more information than if it had not been included in the model. LDL cholesterol is a much more important biomarker. Our second important conclusion addressed the question of whether, after starting a patient on statin therapy, the magnitude of lowering CRP by the statin and the level to which CRP has been reduced predicts cardiovascular outcome. The simple answer from our analyses was that it did not and that the benefits were all related to lowering LDL cholesterol. Our population comprised patients with stable hypertension and no history of coronary disease; likewise, the diabetes population in CARDS had no history of coronary heart disease. Oddly, PROVE IT-TIMI 22 involved persons who were selected from a high-risk coronary heart disease population simply because they had a high level of an inflammatory marker. So, to a certain extent, this is like comparing apples and oranges, and to find some unifying hypothesis on the basis of widely heterogeneous patient populations seems to be a little naive.

[B] Predictor of cardiovascular disease

Acute Phase Reactants as Novel Predictors of Cardiovascular Disease  M. S. AhmedA. B. JadhavA. Hassan, and Qing H. Meng SRN Inflammation 2012; Article ID 953461, 18 pages. doi:10.5402/2012/953461

  • Acute phase reaction is a systemic response which usually follows a physiological condition that takes place in the beginning of an inflammatory process.
  • This physiological change usually lasts 1-2 days. However, the systemic acute phase response usually lasts longer.
  • The aim of this systemic response is to restore homeostasis.
  1. These events are accompanied by upregulation of some proteins (positive acute phase reactants) and
  2. downregulation of others (negative acute phase reactants) during inflammatory reactions.

Cardiovascular diseases are accompanied by the elevation of several positive acute phase reactants such as

  • C-reactive protein (CRP),
  • serum amyloid A (SAA),
  • fibrinogen,
  • white blood cell count,
  • secretory nonpancreatic phospholipase 2-II (sPLA2-II),
  • ferritin, and
  • ceruloplasmin.

Cardiovascular disease is also accompanied by the reduction of important transport proteins such as

  • albumin, transferrin,
  • transthyretin,
  • retinol-binding protein,
  • antithrombin, and
  • transcortin.

In this paper, we will be discussing the biological activity and diagnostic and prognostic values of acute phase reactants with cardiovascular importance.

The potential therapeutic targets of these reactants will be also discussed.

.

[C] CRP as an Inflammatory Biomarker

CRP is an acute phase protein [78] produced in the liver in response to interleukin- (IL-) 6 which is stimulated, in turn, by tumour necrosis factor-α (TNF-α) and IL-1 [89].

Recent studies suggest that CRP plays a pivotal role in many aspects of atherogenesis including

  • LDL uptake by macrophage,
  • release of proinflammatory cytokines,
  • expression of monocyte chemotactic protein-1,
  • intercellular adhesion molecule-1, and vascular cellular adhesion molecule-1 [1012].

Activation of inflammation and the acute phase reaction appear to play an important role, not only in the pathogenesis of atherosclerosis, but also in the initiation of the acute coronary syndrome (ACS) [13,14]. Cesari et al. suggested that the inflammatory markers CRP, IL-6, and TNF-α are independent predictors of cardiovascular events in older persons [14].

Diagnostic Value

CRP is also an early ischemic marker and elevated CRP is predictive of future adverse events [2223]. High-sensitivity CRP (hs-CRP) rises acutely after tissue injury, including myocardial infarction (MI). Intense cytokine production and inflammatory cell infiltration occur in the area of ischemia and necrosis. This increase of hs-CRP levels, in part, correlates with infarct size [2425]

CRP can be also used for patients screening in the primary prevention population [36]. Ockene et al. indicated that CRP is generally expressed at low levels (<1 mg/L) in healthy adults and levels remain relatively stable in the absence of an acute inflammatory stimulus [37].

Patients with unstable angina and CRP >3 mg/L at discharge are more likely to be readmitted for recurrent cardiovascular instability or MI within 1 year [38].

Pietilä et al. indicated that hs-CRP measurement is the strongest correlative factor for future clinical events due to arterial inflammation, myocardial infarction, unstable angina, stroke, and peripheral vascular disease in both diseased and apparently healthy asymptomatic patients [40].

The CRP plasma level also is the best risk assessment in patients with

  • either stable or unstable angina,
  • long term after myocardial infarction, and
  • in patients undergoing revascularization therapies [41].
  • One study showed the only independent cardiovascular risk indicators using multivariate, age adjusted and traditional risk analysis were CRP and Total/HDL cholesterol ratio.
  • If CRP, IL-6, and ICAM-1 levels are added to lipid levels, risk assessment can be improved over lipids alone.
  • Moreover, serum CRP may indicate the vulnerability of the plaque [40].

Prognostic Value

  • elevation of hs-CRP levels predicts a poor cardiovascular prognosis [42].
  • The extent of the inflammatory response to injury appears to have prognostic significance, which is independent of the extent of myocardial injury.
  • hs-CRP response after MI has been shown to predict future CHD morbidity and mortality independent of infarct size [43].
  • CRP is also a predictor of incident type 2 diabetes. As well, it adds a prognostic information on vascular risk at all levels of the metabolic syndrome [44].

__________________________________________________________________________________________________________________________

III.  Troponin(s) and hs-TnI/cTnT

Comparison of diagnostic accuracy between three different rules of interpreting high sensitivity troponin T results. Francesco Buccelletti; Leonarda Galiuto; Davide Marsiliani; Paolo Iacomini; et al. Intern Emerg Med 2012; 7, 365

Abstract

With the introduction of high sensitivity troponin-T (hs-TnT) assay, clinicians face more patients with ‘positive’ results but without myocardial infarction. Repeated hs-TnT determinations are warranted to improve specificity. The aim of this study was to compare diagnostic accuracy of three different interpretation rules for two hs-TnT results taken 6 h apart. After adjusting for clinical differences, hs-TnT results were recoded according to the three rules.

  • Rule1: hs-TnT >13 ng/L in at least one determination.
  • Rule2: change of >20 % between the two measures.
  • Rule3: change >50 % if baseline hs-TnT 14-53 ng/L and >20 % if baseline >54 ng/L.

The sensitivity, specificity and ROC curves were compared. The sensitivity analysis was used to generate post-test probability for any test result. Primary outcome was the evidence of coronary critical stenosis (CCS) on coronary angiography in patients with high-risk chest pain.

183 patients were analyzed (38.3 %) among all patients presenting with chest pain during the study period. CCS was found in 80 (43.7 %) cases.

The specificity was 0.62 (0.52-0.71), 0.76 (0.66-0.84) and 0.83 (0.74-0.89) for rules 1, 2 and 3, respectively (P < 0.01). Sensitivity decreased with increasing specificity (P < 0.01).

Overall diagnostic accuracy did not differ among the three rules (AUC curves difference P = 0.12). Sensitivity analysis showed a 25 % relative gain in predicting CCS using rule 3 compared to rule 1. Changes between two determinations of hs-TnT 6 h apart effectively improved specificity for CCS presence in high-risk chest pain patients.

There was a parallel loss in sensitivity that discouraged any use of such changes as a unique way to interpret the new hs-TnT results.

Advances in identifying NSTEMI biomarkers [Published 31 August 2012 | Article by Excerpta Medica | Tags: elderly, ami, biomarkers, diagnosis]

In the run-up to the ESC conference at the end of August, we review some recently published research on the hot topic of biomarkers for NSTEMI.

Prompt and accurate diagnosis of acute non-ST elevation myocardial infarction (NSTEMI) can be particularly challenging in elderly patients, as they often present with

  • atypical symptoms and/or have an inconclusive ECG.
  • the diagnostic value of cardiac troponin T (cTnT), an established marker of cardiac injury, is often limited as there is often non-coronary troponin elevation caused by concomitant conditions such as acute congestive heart failure.
  • Identifying new sensitive and specific biomarkers of NSTEMI in elderly patients is therefore important, and circulating microRNAs (miRs) are emerging as good candidates.

researchers evaluated the diagnostic potential of plasma levels of various miRs in elderly patients enrolled at presentation:

  • 92 acute NSTEMI patients (complicated by congestive heart failure in three-quarters of cases),
  • 81 patients with acute congestive heart failure without acute MI, and
  • 99 age-matched healthy people (the control group).

The researchers, from centers in Italy, found that levels of miR-1, miR-21, miR-133a, and miR-423-5p were 3-10 times higher in the patients with NSTEMI, compared with controls. Levels of miR-499-5p, meanwhile, were more than 80 times higher in the NSTEMI patients compared with the patients in the control group.

  • Levels of miR-499-5p and miR-21 were also significantly higher in the NSTEMI group compared with the group of patients with acute congestive heart failure without acute MI.
  • The researchers also found that miR-499-5p was similar to cTnT in being able to distinguish NSTEMI patients from the other two groups.
  • Also, a subgroup analysis of patients with a modest elevation in cTnT level at presentation (0.03-0.10 ng/mL) revealed that miR-499-5p had a diagnostic accuracy superior both to cTnT and to high sensitivity cTnT in differentiating NSTEMI from acute congestive heart failure.

International Journal of Cardiology

________________________________________________________________________________________________________________________________________________________________________

IV. Predicting cardiovascular mortality in NSTEMI patients

[A]  Japanese researchers studied 258 consecutive patients hospitalized for unstable angina and NSTEMI within 24 hours of the onset of chest symptoms, and followed them up for a median period of 49 months after admission. During this period there were 38 cardiovascular deaths (14.7%).

They reported that high-mobility group- box 1 (HMGB1), a nuclear protein and signaller of tissue damage, was “a potential and independent predictor of cardiovascular mortality in patients hospitalized for unstable angina and NSTEMI.

  • HMGB1,
  • cardiac troponin I,
  • Killip class greater than 1, and
  • age

were each independently and significantly associated with cardiovascular mortality.

……………………………………………………………………………………………………………………………………………………………………………………………………………………………………………………………………………………………………

[B]  William LaFramboise et al.       BMC Med. 2012 Dec 5;10(1):157)
see Report by Aviva Lev-Ari (pharmaceuticalintelligence.com)  Coronary artery disease in symptomatic patients referred for coronary angiography: Predicted by Serum Protein Profiles

Significant differences were detected in circulating proteins from patients requiring revascularization including increased apolipoprotein B100 (APO-B100), C-reactive protein (CRP), fibrinogen, vascular cell adhesion molecule 1 (VCAM-1), myeloperoxidase (MPO), resistin, osteopontin, interleukin (IL)-1beta, IL-6, IL-10 and N-terminal fragment protein precursor brain natriuretic peptide (NT-pBNP) and decreased apolipoprotein A1 (APO-A1). Biomarker classification signatures comprising up to 5 analytes were identified using a tunable scoring function trained against 239 samples and validated with 120 additional samples. A total of 14 overlapping signatures classified patients without significant coronary disease (38% to 59% specificity) while maintaining 95% sensitivity for patients requiring revascularization. Osteopontin (14 times) and resistin (10 times) were most frequently represented among these diagnostic signatures. The most efficacious protein signature in validation studies comprised osteopontin (OPN), resistin, matrix metalloproteinase 7 (MMP7) and interferon gamma (IFNgamma) as a four-marker panel while the addition of either CRP or adiponectin (ACRP-30) yielded comparable results in five protein signatures.

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V.  Assessing Cardiovascular Risk

Agency for Healthcare Research and Quality (AHRQ)

Assessing Cardiovascular Risk: Guideline Synthesis

Agency for Healthcare Research and Quality (AHRQ) Authors and Disclosures Posted: 03/01/2012

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The Third MI Definition: An Expert Interview With Joseph Alpert In the new definition, the diagnosis of acute MI remains unchanged: That is, it applies where there is evidence of myocardial necrosis in a clinical setting consistent with acute myocardial ischemia. However, the criteria for diagnosis have been updated, with an emphasis on the biomarker cardiac troponin.

  1. The first essential criterion for diagnosis of MI is detection of a rise or fall in cardiac troponin, or CKMB if troponin is not available, with at least 1 value above the 99th percentile upper reference limit, plus at least 1 the following criteria:
  2. Symptoms of ischemia;
  3. ECG changes of new or presumed new ischemia (significant ST-segment T-wave changes or new left bundle branch block);
  4. Development of pathologic Q waves on ECG; or
  5. Imaging evidence of new loss of viable myocardium or new regional wall-motion abnormality.Other criteria include those for MI in sudden unexpected cardiac death and for MI during percutaneous coronary intervention (PCI) and coronary artery bypass graft surgery (CABG).

The guidance document supports the use of high-sensitivity cardiac troponin assays, especially for distinguishing myocardial injury not related to myocardial ischemia, such as that associated with heart failure or renal failure. These assays are available in Europe, and not in the United States. MI is designated as ST-segment elevation MI or non- ST-segment elevation MI, and as in the 2007 version, it is classified into 5 types on the basis of pathologic, clinical, and prognostic differences. These types have been updated in the latest version.

  1. Type 1 MI (spontaneous MI) is related to atherosclerotic plaque rupture or other event leading to thrombus formation in ≥ 1 of the coronary arteries, leading to decreased myocardial blood flow with ensuing necrosis;
  2.  Type 2 MI arises from a condition other than CAD;
  3.  Type 3 MI is deemed to have occurred when cardiac death occurs with symptoms suggestive of myocardial ischemia, but without biomarker values having been obtained; and
  4. Type 4 and 5 MIs are related to PCI and CABG, respectively, and have been redefined since 2007.

The new document also describes situations in which troponin levels are elevated in conditions where myocardial injury with necrosis is associated with predominantly nonischemic myocardial injury, such as heart failure, renal failure, myocarditis, arrhythmias, or pulmonary embolism.

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VI Statistical Problems

The normal or “bell shaped” curve is a plot of numerical values along the x-axis and the frequency of the occurrence on the y-axis.  If the set of measurements occurs as a random and independent event, we refer to this as parametric, and the distribution of the values is a bell shaped curve with all but 2.5% of the values included within both ends, with the mean or arithmetic average at the center, and with 67% of the sample contained within 1 standard deviation of the mean.  We view a reference range in terms of a homogeneous population.  This means that while all values might not be the same, the values are scattered within a distance from the mean that becomes less frequent as the distance is larger so that we can describe a mean and a 95% confidence interval around the mean.  In the problem we are discussing, the classic reference value could be determined with outliers removed, and it would most likely fit to a Receiver Operator Characteristic curve.  This became blurred when the high sensitivity assay was introduced, which included NOISE, which is really not noise but heterogeneous elements related to [a] vascular events that are not caused by plaque rupture, or [b] ischemia related to “piecemeal necrosis” which continued might predict a serious future event.  Hidden variables include – age, diurnal variation, racial factors, and disease (hypertension, CHF, type 2 diabetes, renal failure).

A majority have no ST elevation on EKG, considered definitive for AMI.  This makes the finding of elevated and increasing cardiac specific enzyme or protein in serum of paramount importance for specifying damaged cardiac muscle in a context of insufficient circulation.   We examine the classification of AMI using a combination of features of chest pain, EKG, and a sensitive cardiac marker, derived from the cardiac muscle filament.   An optimum value for a test cutoff is, such as cTnT, can be derived without using a prior determination of disease status. This is an alternative to first carrying out a study with a training set, and then repeating it with a validation set, provided there is sufficient information for classifying the data..  We have to construct a self-classifying table of ordered classes that have assigned measurable risks.   Haberman (4) discusses the underlying assumptions used by Magidson for association models of cross-classified data in calculating the maximum likelihood estimates (MLE) by using the log-likelihood ratio and a sum of squares representing deviations of parameters from their constraints. The Latent Class Analysis (LCA) developed by Magidson and Vermunt allows use of a traditional LCA or a regression model (Statistical Innovation. Belmont, MA).  .  The LCA model uses the variables – chest pain, EKG, and troponin T – to classify the data and to test the underlying structure using powerful fit measures, such as L2.  Chest pain has the value of “typical” or “other”.   EKG has the value ST depression or any other (for a non Q-wave study).  “cTnT” has the value  assigned by rank in the scaling intervals.   The results of such an analysis are displayed in Table 1.

Table 1. This LCA classification was constructed using the troponin T before hsTnT was available.

CTnT (mg/L) MI (6%)  Not MI (94%)
0-0.03 0.0008 0.8485
0.031-0.055 0.0009 0.0791
0.056-0.080 0.0009 0.0369
0.081-0.099 0.0010 0.0106
0.10-0.199 0.2026 0.0238
> 0.20 0.7939 0.0012

Eugene Rypka. Syndromic classification: A process for amplifying information using S-Clustering.  Nutrition 1996; 14(12: 827-829.

Stuart W Zarich, Keith Bradley, Inder Dip Mayall, Larry H Bernstein. Minor elevations in troponin T values enhance risk assessment in emergency department patients with suspected myocardial ischemia: analysis of novel troponin T cut-off values. Clin Chim Acta 2004; 343(1-2):223-229

Haberman SJ. Computation of maximum likelihood estimates in association models. J Am Stat Assoc 1995;90:1438-1446

Rudolph RA, Bernstein LH, Babb J.  Information Induction for the Diagnosis of  Myocardial Infarction. Clin Chem 1988; 34: 2031-8.

Vermoent JK, and Magidson J. Latent class cluster analysis. JA Hagenaars and AL McCutcheon (eds.), Advanced Latent Class Analysis. Cambridge, Cambridge University Press, 2000.

Bernstein LH, Qamar A, McPherson C, Zarich S. Evaluating a new graphical ordinal logit method (GOLDminer) in the diagnosis of myocardial infarction utilizing clinical features and laboratory data.   Yale J Biol Med 1999; 72: 259-268.

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VII. Conclusions I have made a number of comments to follow up on.  The diagnosis of myocardial infarct has been extended as a result of the emergence of the high sensitivity troponins, but the laboratory methods have not caught up with the technology as the identification of myocardial ischemia is broken down with fine granularity in order to

  • identify the high risk patients early
  • and manage them effectively at the earliest stage of disease evolution

We no longer ponder over

  1. ECG findings of new Q-qave, not previously seen
  2. ST elevation
  3. T-wave inversion

These are an indication of plaque rupture. There are strong associations between CRP, hyperhomocysteinemia, and then add the troponins and b-type natriuretic  and the pro b-type peptides.  These associations have to be analyzed by “syndromic classification”, described by Eugene Rypka. The study first described found great value in the BNP and proBNP.  Despite having the creatinine clearance, the NT-proBNP can’t be adequately interpreted without adjusting for the estimated glomerular filtration rate, and using a log transform for the high fold-increase with age.

There is much more to be done with capturing the information from the data we are generating.  The problem of classification requires accurate data measurement, but it also requires that features in scaled data are combined in meaningful ways.  That job is far from completed.

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Below I paste all discussions on this post that are taking place on LinkedIn Group: Innovations in Cardiology:

Kathy Dowd, AuD • I am an audiologist representing the Academy of Doctors of Audiology for an initiative of early identification of hearing loss in adults with chronic diseases, including cardiovascular disease, thyroid disease, diabetes, etc. I am working on a new product that will automatically screen hearing of patients with these conditions and route them to audiologists for evaluation, treatment and counseling. Hearing loss is unidentified for most adults for 7-10. The psychological impact of hearing loss includes depression, isolation, confusion and poor job performance. We are focused on educating healthcare professionals on the need to identify this ‘silent epidemic’ as a co morbidity with most major illnesses.

Aviva Lev-Ari, PhD, RN • Dr. William LaFramboise

Thank you for your comment above and the reference to your most recent publication. It is very helpful. We are on the same page on this topic.

May I bring to the attention of the readers three sources which are shading additional light on that matter.

To Stent or Not? A Critical Decision

To Stent or Not? A Critical Decision

Obstructive coronary artery disease diagnosed by RNA levels of 23 genes – CardioDx heart disease test wins Medicare coverage

http://pharmaceuticalintelligence.com/2012/08/14/obstructive-coronary-artery-disease-diagnosed-by-rna-levels-of-23-genes-cardiodx-heart-disease-test-wins-medicare-coverage/http://pharmaceuticalintelligence.com/?s=PCI

Follow William
William LaFramboise • Thank you Aviva. The CardioDx approach is promising with heavy commercial support for use in a primary care practitioner’s office. However, RNA acquisition, purification and qRT-PCR expression analysis takes 2-3 days, is performed off-site, derives from a small subset of circulating inflammatory cells and is not yet directly correlated with functional proteomics. So its value in the Emergency Room and Chest Clinic is currently limited. The proteomics test we published revealed systemic serum changes in a small number of proteins known to be involved in atherosclerosis. It has a faster turnaround time (minutes to hours) that could be implemented in an emergency room or chest clinic. We are predominantly interested in using our test to “rule out” symptomatic patients who currently undergo coronary angiography but do NOT have clinically significant CAD. Our goal is to eliminate unecessary catheterizations while catching all patients that should undergo coronary angiography with a high probability of percutaneous intervention. Currently, the patients we are targeting all undergo catheterization; our test will hopefully allow us to identify at least some of these patients who do not have CAD and eliminate this expensive and risky procedure. However, we are in the pioneering stages of developing our test so there are miles to go before we establish and validate clinical efficacy.

Larry

Larry Bernstein • What you have indicated is practical proteomics. There is more to be done in line with what Dr Lev-Ari has indicated based on additional voluminous literature. What you have done with a not so large data set, and probably underpowered looks very interesting.

I f you were willing to share the data, now that it is publihed, I think that we can sharpen the results using a method of “identifying anomalies”, and even come up with estimated probabilities for meaningful classes. I think that the best you can get is defined by Kullback-Liebler distance.

Larry H Bernstein, MD
larry.bernstein@gmail.com

Biomarker classification signatures comprising up to 5 analytes were identified using a tunable scoring function trained against 239 samples and validated with 120 additional samples. A total of 14 overlapping signatures classified patients without significant coronary disease (38% to 59% specificity) while maintaining 95% sensitivity for patients requiring revascularization. Osteopontin (14 times) and resistin (10 times) were most frequently represented among these diagnostic signatures. The most efficacious protein signature in validation studies comprised osteopontin (OPN), resistin, matrix metalloproteinase 7 (MMP7) and interferon gamma (IFNgamma) as a four-marker panel while the addition of either CRP or adiponectin (ACRP-30) yielded comparable results in five protein signatures.

Proteins in the serum of CAD patients predominantly reflected (1) a positive acute phase, inflammatory response and (2) alterations in lipid metabolism, transport, peroxidation and accumulation.

Follow William
William LaFramboise • Our study (http://www.ncbi.nlm.nih.gov/pubmed/23216991) comprised discovery research using targeted immunochemical screening of retrospective patient samples using both Luminex and Aushon platforms as opposed to shotgun proteomics. Hence the costs constrained sample numbers. Nevertheless, our ability to predict outcome substantially exceeded available methods:

DISCUSSION
The Framingham CHD scores were statistically different between groups (P <0.001, unpaired Student’s t test) but they classified only 16% of the subjects without significant CAD (10 of 63) at a 95% sensitivity for patients with CAD. In contrast, our algorithm incorporating serum values for OPN, RES, CRP, MMP7 and IFNγ identified 63% of the subjects without significant CAD (40 of 63) at 95% sensitivity for patients with CAD. Thus, our multiplex serum protein classifier correctly identified four times as many patients as the Framingham index.

The addition of clinical variables to our scoring system should improve the acuity of our test as we move into the next phase. I appreciate your input and will contact you directly for further insights

Larry Bernstein • Bill La Fram..

our algorithm incorporating serum values for OPN, RES, CRP, MMP7 and IFNγ identified 63% of the subjects without significant CAD (40 of 63) at 95% sensitivity for patients with CAD

I think you can improve the algorithm with strong clinical features. The Goldman algorithm is stronger than the Framingham Index. Maybe its because the FI is epidemiological and is a measure of long term risk being present and does not indicate significant features at the time of presenting. The best features of the Goldman algorithm (without lab work) are – ECG (which may be arguable with NSEMI), but the presence of Afib or tachyarrhythmia could be added to that in weighting, and radiation actually should include symptoms of gall bladder (vagal nerve branch), and onset, characteristic and duration of pain, and SOB.

In your algorithm there isn’t any assessment of the hypercoagulable state, blood flow or Viscosity. There is a strong relationship between hyperhomocysteinemia and CVD, and the HHCys has ties to impaired methyl group transfers that maybe proinflammatory through more than one interaction: countering eNOS, related to Lp(a), un unknown relationship to iNOS (which becomes a counterpoise to eNOS), an effect on blood flow and viscosity, and a relationship to platelet aggregation.

Lp(a) was originally considered of less weight, except that it occurred in thin people from Asian Indian descent. The relationship to apo(B) and to dense LDL particles is now a factor. Sam Filligane uses Lp(a) in his ambulatory practice, and he also uses the PLAC test that Aviva has posted on.

The biggest problem we have is the amount of variability in the data physicians use. It makes metaanalysis a poor solution to the problem. The standardization of laboratory “panels” set up after CLIA 88 puts a real burden on the physician for unsubstantiated “cost benefits” in the light of today’s knowledge.

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English: Four chamber view on cardiovascular m...

English: Four chamber view on cardiovascular magnetic resonance imaging. (Photo credit: Wikipedia)

Other related articles on Assessing Cardiovascular Disease with Biomarkers published on this Open Access Online Scientific Journal, include the following:

 

Dr. Lev-Ari’s research on Assessing Cardiovascular Disease with Biomarkers includes

  • Biomarkers in vascular biology,
  • Biomarkers in molecular cardiology and
  • circulating Endothelial Progenitor Cells (cEPCs) as a Biomarker for cardiovascular marcovascular disease risk

 

Lev-Ari, A., (2012U). Cardiovascular Outcomes: Function of circulating Endothelial Progenitor Cells (cEPCs): Exploring Pharmaco-therapy targeted at Endogenous Augmentation of cEPCs

http://pharmaceuticalintelligence.com/2012/08/28/cardiovascular-outcomes-function-of-circulating-endothelial-progenitor-cells-cepcs-exploring-pharmaco-therapy-targeted-at-endogenous-augmentation-of-cepcs/

Lev-Ari, A., (2012T). Endothelial Dysfunction, Diminished Availability of cEPCs, Increasing CVD Risk for Macrovascular Disease – Therapeutic Potential of cEPCs

http://pharmaceuticalintelligence.com/2012/08/27/endothelial-dysfunction-diminished-availability-of-cepcs-increasing-cvd-risk-for-macrovascular-disease-therapeutic-potential-of-cepcs/

Lev-Ari, A., (2012S). Vascular Medicine and Biology: CLASSIFICATION OF FAST ACTING THERAPY FOR PATIENTS AT HIGH RISK FOR MACROVASCULAR EVENTS Macrovascular Disease – Therapeutic Potential of cEPCs

http://pharmaceuticalintelligence.com/2012/08/24/vascular-medicine-and-biology-classification-of-fast-acting-therapy-for-patients-at-high-risk-for-macrovascular-events-macrovascular-disease-therapeutic-potential-of-cepcs/

Lev-Ari, A. (2012a). Resident-cell-based Therapy in Human Ischaemic Heart Disease: Evolution in the PROMISE of Thymosin beta4 for Cardiac Repair

http://pharmaceuticalintelligence.com/2012/04/30/93/

Lev-Ari, A. (2012b). Triple Antihypertensive Combination Therapy Significantly Lowers Blood Pressure in Hard-to-Treat Patients with Hypertension and Diabetes

http://pharmaceuticalintelligence.com/2012/05/29/445/

Lev-Ari, A. (2012h). Macrovascular Disease – Therapeutic Potential of cEPCs: Reduction Methods for CV Risk

http://pharmaceuticalintelligence.com/2012/07/02/macrovascular-disease-therapeutic-potential-of-cepcs-reduction-methods-for-cv-risk/

Lev-Ari, A. (2012xx). Coronary artery disease in symptomatic patients referred for coronary angiography: Predicted by Serum Protein Profiles

http://pharmaceuticalintelligence.com/2012/12/29/coronary-artery-disease-in-symptomatic-patients-referred-for-coronary-angiography-predicted-by-serum-protein-profiles/

Lev-Ari, A. (2013a) forthcoming, based on:

Part III: (2006c) Biomarker for Therapeutic Targets of Cardiovascular Risk Reduction by cEPCs Endogenous Augmentation using New Combination Drug Therapy of Three Drug Classes and Several Drug Indications. Northeastern University, Boston, MA 02115

Special Considerations in Blood Lipoproteins, Viscosity, Assessment and Treatment                                    Larry Bernstein

New Insights on Nitric Oxide donors – Part IV                Larry Bernstein

The Essential Role of Nitric Oxide and Therapeutic NO Donor Targets in Renal Pharmacotherapy             Larry Bernstein

A second look at the transthyretin nutrition inflammatory conundrum                                                                  Larry Bernstein

What is the role of plasma viscosity in hemostasis and vascular disease risk?                                                        Larry Bernstein

Biochemistry of the Coagulation Cascade and Platelet Aggregation – Part I                                                            Larry Bernstein

Laboratory, Innovative Technology, Therapeutics                                                                                                            Larry Bernstein

Ubiquinin-Proteosome pathway, autophagy, the mitochondrion, proteolysis and cell apoptosis                 Larry Bernstein

Overview of new strategy for treatment of T2DM: SGLT2 inhibiting oral antidiabetic agents                             aviralvatsa

Mitochondrial dynamics and cardiovascular diseases          ritusaxena

Nitric Oxide and it’s impact on Cardiothoracic Surgery        tildabarliya

Telling NO to Cardiac Risk                                                                  sjwilliamspa

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Carotid Stenting: Vascular surgeons have pointed to more minor strokes in the stenting group and cardiologists to more myocardial infarctions in the CEA cohort.

Reporter: Aviva Lev-Ari, PhD,RN

Why CREST is a Game Changer for Carotid Stenting

 

Speaker: Gary Roubin, MD, PhD

The CREST Trial was the largest, most rigorous and because of the way it was conducted, the most relevant investigation into the role of carotid stenting to date.

The National Institute of Neurological Diseases after critical review concluded that the study “Demonstrated that Endarterectomy and Stenting were equally efficacious methods of preventing stroke caused by carotid bifurcation stenoses.”

The primary endpoint was unequivocal but the components of this combined endpoint have been dissected by various groups to support different conclusions. Vascular surgeons have pointed to more minor strokes in the stenting group and cardiologists to more myocardial infarctions in the CEA cohort. The CREST Trial demonstrated remarkable safety from both procedures with a very low and similar major stroke and death rate.  The small numbers of excess strokes in the stenting group were minor strokes and importantly further analyses of temporal trends have demonstrated this delta disappeared over the course of the study.  Stenting stroke rates improved over time probably related to better selection of younger patients with more suitable anatomy for stenting.

If CREST was to restart in 2012, it is extremely unlikely that any difference whatsoever would be seen in comparing CEA and Stenting.

Importantly, minor strokes were not associated with a later excess mortality while a periprocedural MI was associated with death over the follow up period.

Quality of life analyses reflected the minor, non disabling nature of the small number of excess minor strokes.  The comprehensive panel of SF36 mental and physical quality of life measures demonstrated no difference whatsoever between stenting and CEA.

Despite completing the study with first generation stents and embolic protection devices, the outcomes were gratifying.  A critical FDA panel subsequently approved the extension of labeling for stenting use in standard risk CEA patients.

Now we await a considered response from CMS to acknowledge the demonstration of “reasonable safety and efficacy” and long awaited reimbursement for this patient friendly, percutaneous procedure.

We now are experiencing a curious push back from some in the neurological community and even some surgeons who argue that neither CEA nor stenting are needed in the treatment of asymptomatic patients. 

  • This—despite multiple trials that have demonstrated the superiority of revascularization and markedly improved revascularization results.
  • This—despite no scientific evidence to support the equivalence of medical therapy in preventing stroke in carotid bifurcation disease.

 

Panel Discussion

 

NICK HOPKINS

The CREST data stand on their own merits. Looking at the survival curves for minor stroke versus myocardial infarction, as a surgeon who does a large volume of CEA and stenting, I am impressed with the benign outcome in the minor strokes and the bad outcomes associated with M.I. Again, as a surgeon, I doubt we can do anything to reduce the incidence of MI, but as a stent operator, I feel we can do much more to further reduce the incidence of stroke events.

For example, the neurological community has focused on the ICSS Trial sub-study that demonstrated a significant incidence of MRI-DWI defects after stenting. We don’t really know what DWI changes mean but the neurological community assumes they are bad.  We see a 15-20% incidence with just a routine angiogram and they are probably just micro-bubbles causing these temporary defects. In the ICSS trials, the incidence of these lesions was 50% in the stent arm and 12% in the CEA arm.   The conclusion was that stenting was “bad” but embolic protection devices were only used in 75% of the ICSS patients. Now we have new devices such as proximal occlusion balloons that have been shown to markedly reduce the incidence of these lesions. So, this is just one example of new stenting techniques that will reduce the incidence of stroke to even lower rates. There is also a lot of activity in the industry to make carotid stents covered with a fine, semi-permeable membrane that will reduce the chance of embolic debris from the procedure. With current devices and certainly the stent used in CREST, debris may be forced through the stent strut when you dilate.

So to me these are just two examples of things that will improve the stroke rate from the current 1% to 3% to near zero.

TY COLLINS

I am not sure what happened at the Medicare Coverage Advisory Meeting last week (January 2012) but basically the committee did not appear to focus on the CREST data.

JIRI VITEK

One of the most important differentiating features of CREST compared to the European Trials was the emphasis on operator credentialing and ongoing training of operators over the 8 years of recruitment.  This is evidenced by the improvement of stent outcomes over the time course of the trial.

I also want to point out that none of these trials place enough emphasis on cranial nerve injuries that are an exclusive and important complication of CEA.  In CREST there was a 4.5% incidence of cranial nerve palsy in the CEA cohort, and 2% were still present at 6 months.

BARRY KATZEN

I firmly believe that CREST is a “game changer.” I spent a full day at the Medicare Coverage Advisory Committee last week (January 2012). Two things happened. The first was that the entire discussion was derailed by irrelevant discussion of the supposed value of medical therapy. As Ty said before, nobody appeared to be focused on the CREST data but was distracted by arguments about the value of medical management. A neurologist, Anne Abbott, took a large amount of time basically “trouncing” any type of revascularization therapy. 

The critical consideration by the FDA and their approval of the devices for this indication is a better representation of where we stand today. Interdisciplinary factional disputes and politics aside, I believe CMS will want to expand the coverage for carotid stenting in some way.

JIM ZIDAR

I will say that although CREST is a “game changer”, it seems the cards may be stacked against stenting.  Besides the cost issues that may be associated with expanded coverage, they are influenced by the self interest of the Society of Vascular Surgery that decided they were not going to support the data. If coverage is not expanded for stenting, I wonder if it is not unreasonable for other professional societies to conclude and pronounce that CEA should not be reimbursed in asymptomatic patients.

NICK HOPKINS

CMS would be interested in that.  I actually think that given the dialogue on the day, it definitely could have gone that way.

BARRY KATZEN

It is fascinating to think about this. Given all of the level 1 scientific data supporting revascularization over medical therapy, in the United States today, CEA is the standard of care. Primary care physicians throughout the country recommend this for patients with severe stenoses. CMS is basically talking about erasing that standard of care or now an equivalent procedure from a reimbursement standpoint.

GARY ROUBIN

Let us all be clear about the evidence from CREST. In the younger patients, male patients and asymptomatic patients there was no significant difference in outcomes for stroke and death between stenting and CEA. 

 

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Evidence for Overturning the Guidelines in Cardiogenic Shock

Reporter: Aviva Lev-Ari, PhD, RN

 

Christopher M. O’Connor, M.D., and Joseph G. Rogers, M.D.

August 27, 2012 (10.1056/NEJMe1209601)

Cardiogenic shock complicating acute myocardial infarction continues to be a devastating event associated with extremely high mortality. The results of the Intraaortic Balloon Pump in Cardiogenic Shock II (IABP-SHOCK II) trial,1 now reported in the Journal, show that in patients with acute myocardial infarction and hemodynamic compromise who undergo revascularization, the routine use of an intraaortic balloon pump (IABP), as compared with standard therapy, does not improve survival.1

Introduced nearly five decades ago,2 IABP is now routinely used as an adjuvant treatment for myocardial infarction complicated by cardiogenic shock, on the basis of evidence that it is associated with hemodynamic improvements accompanied by enhanced coronary blood flow, increased perfusion of vital organs, maintenance of infarct-artery patency, and decreased systemic inflammation.3 Until recently, few alternatives have been available to support patients with severely compromised hemodynamics. Despite a lack of robust data from outcomes trials and meta-analyses4,5 that have shown limited efficacy, international guidelines endorse the use of IABP for treating post-myocardial infarction shock, with a class I recommendation.6,7

The IABP-SHOCK II trial could have affirmed contemporary clinical practice and guidelines. Instead, it revealed surprising results. In a comparison of IABP with standard therapy, the investigators found no difference in 30-day mortality or in any key secondary end points. Although IABP was safe, there was no evidence that it was associated with hemodynamic improvement — a mechanistic effect of IABP that has long been considered to be critical for its clinical application. Moreover, there were no benefits with respect to renal function or attenuation in lactate or C-reactive protein levels.

Conducting a randomized clinical trial in the emergency setting of acute myocardial infarction with shock is exceptionally difficult. In an era of rapid intervention in the catheterization laboratory and a pervasive perception of lack of equipoise, the IABP-SHOCK II investigators should be commended for completing a moderate-sized trial in 3 years at 37 sites. The results of the trial have important clinical implications, but several issues must be addressed. First, this 600-patient study could be considered too small to be definitive. However, the conclusions are bolstered by more than 240 primary end-point events, making them far more robust than might be surmised from the sample size alone. Second, the patients represented a moderate-risk cohort, with a 30-day mortality of 40%. This rate is lower than that reported in other trials involving patients with cardiogenic shock, so the results may not be applicable to the highest-risk patients. In the decade since the original Should We Emergently Revascularize Occluded Coronaries for Cardiogenic Shock (SHOCK) trial was reported,8 emphasis on early revascularization and increased use of background medical therapies would be anticipated to have favorably altered the natural history of cardiogenic shock occurring after myocardial infarction, leading to lower 30-day mortality. Third, asymmetry in the number of crossovers may have influenced the final results analyzed according to the intention-to-treat principle. However, a per-protocol analysis (which included all the patients who had confirmed acute myocardial infarction, with the exclusion of those who crossed over) and an adjusted multivariate model showed similar nonsignificant results. Finally, favorable trends with IABP were observed in younger patients and in those with a first myocardial infarction, although these findings can be considered only as hypothesis-generating. Given the concordance of data from the meta-analyses and the current trial, the data do not support the routine use of IABP in patients with acute myocardial infarction complicated by cardiogenic shock, and the level I guideline recommendation is now strongly challenged. Members of guideline committees and clinicians should take note of another example of a recommendation that is based on insufficient data.

The results of the IABP-SHOCK II trial parallel those from many recent outcome trials that have challenged our understanding of the management of acute and chronic heart failure, including those regarding the use of pulmonary artery catheters9 and the role of revascularization in ischemic cardiomyopathy.10

Therapeutic strategies for patients with cardiogenic shock have changed abruptly and are ready for renewed growth and development. Although many will find the results of the IABP-SHOCK II trial disappointing, we must recognize the opportunity to develop novel and innovative strategies to treat this condition. Integrated systems to ensure rapid reperfusion may reduce the incidence of shock among patients who have had an acute myocardial infarction.11 Secondary analyses of data from the IABP-SHOCK II trial may help us understand the mechanisms of the failed response. Comparing the patient populations and outcomes of the IABP-SHOCK II study groups and the concurrent registry cohort may yield important insights, with therapeutic implications for the use of other mechanical devices for circulatory support. On the basis of the findings of the IABP-SHOCK II trial, we must move forward with the understanding that a cardiovascular condition with 40% mortality at 30 days remains unacceptable. Most important, we hope that the results of this trial will galvanize a broadly based mandate to address this devastating clinical problem by reestablishing equipoise and international engagement in research on novel devices and pharmacologic therapies.

This article was published on August 27, 2012, at NEJM.org.

Source Information

From Duke University, Durham, NC.

http://www.nejm.org/doi/full/10.1056/NEJMe1209601

 

Intraaortic Balloon in Cardiogenic Shock 

Original Article

Intraaortic Balloon Support for Myocardial Infarction with Cardiogenic Shock

H. Thiele and Others

In current international guidelines, intraaortic balloon counterpulsation (IABP) is considered to be a class I treatment for cardiogenic shock complicating acute myocardial infarction. However, evidence is based mainly on registry data, and there is a paucity of randomized clinical trials.

Clinical Pearls

  What were the results of this study, which compared intraaortic balloon counterpulsation or no intraaortic balloon counterpulsation in patients with cardiogenic shock complicating acute myocardial infarction?

At 30 days, mortality was similar among patients in the IABP group and those in the control group (39.7% and 41.3%, respectively; relative risk with IABP, 0.96; 95% confidence interval, 0.79 to 1.17; P=0.69).

Table 3.Clinical Outcomes.

Figure 1. Time-to-Event Curves for the Primary End Point.

  Among patients who received IABP, did results differ between those who had the IABP inserted before versus after revascularization?

Among patients in the IABP group, there was no significant difference in mortality between the patients (13.4%) in whom the balloon pump was inserted before revascularization and the patients (86.6%) in whom the balloon pump was inserted after revascularization (mortality, 36.4% and 36.8%, respectively; P=0.96).

Morning Report Questions

Q. Did safety end points differ between the two groups? 

A. There were no significant differences between the IABP group and the control group with respect to the rates of stroke, bleeding, sepsis, or peripheral ischemic complications requiring intervention in the hospital. There were also no significant differences in the rates of reinfarction or stent thrombosis.

Q. How do the authors explain the effect of intraaortic balloon counterpulsation in this trial on the factors that are known to cause death in patients with cardiogenic shock? 

A. Death in patients with cardiogenic shock can result from one or more of three factors: hemodynamic deterioration, occurrence of multiorgan dysfunction, and development of the systemic inflammatory response syndrome. There was no immediate improvement in blood pressure or heart rate among patients in whom an intraaortic balloon pump was inserted, as compared with those who did not have a balloon pump inserted. Although there was a positive effect of intraaortic balloon counterpulsation on multiorgan dysfunction at day 2 and day 3, this effect was not evident at day 4. There were also no significant effects on C-reactive protein level or serum lactate level, which were assessed as measures of inflammation and tissue oxygenation. Experimental and clinical studies have indicated that intraaortic balloon counterpulsation results in a hemodynamic benefit as a result of afterload reduction and diastolic augmentation with improvement in coronary perfusion. The authors postulate that the effects on cardiac output are modest and might not be sufficient to reduce mortality.

 source:

NEJM Resident E-Bulletin <resebulletin@nejm.org> on 10/3/2012

References

    1. 1Thiele H, Zeymer U, Neumann F-J, et al. Intraaortic balloon support for myocardial infarction with cardiogenic shock. N Engl J Med 2012. DOI: 10.1056/NEJMoa1208410.

    1. 2Kantrowitz A, Tjonneland S, Freed PS, Phillips SJ, Butner AN, Sherman JL Jr. Initial clinical experience with intraaortic balloon pumping in cardiogenic shock. JAMA 1968;203:113-118
      CrossRef | Web of Science | Medline

    1. 3Ohman EM, George BS, White CJ, et al. Use of aortic counterpulsation to improve sustained coronary artery patency during acute myocardial infarction: results of a randomized trial. Circulation 1994;90:792-799
      CrossRef | Web of Science

    1. 4Sjauw KD, Engstrom AE, Vis MM, et al. A systematic review and meta-analysis of intra-aortic balloon pump therapy in ST-elevation myocardial infarction: should we change the guidelines? Eur Heart J 2009;30:459-468
      CrossRef | Web of Science

    1. 5Unverzagt S, Machemer MT, Solms A, et al. Intra-aortic balloon pump counterpulsation (IABP) for myocardial infarction complicated by cardiogenic shock. Cochrane Database Syst Rev 2011;7:CD007398-CD007398

    1. 6Antman EM, Anbe DT, Armstrong PW, et al. ACC/AHA guidelines for the management of patients with ST-elevation myocardial infarction: a report of the American College of Cardiology/American Heart Association Task Force on Practice Guidelines (Committee to Revise the 1999 Guidelines for the Management of Patients with Acute Myocardial Infarction). Circulation 2004;110:e82-e292[Erratum, Circulation 2005;111:2013-4, 2007;115(5):e411, 2010;121(23):e441.]
      CrossRef | Medline

    1. 7Van de Werf F, Bax J, Betriu A, et al. Management of acute myocardial infarction in patients presenting with persistent ST-segment elevation. Eur Heart J 2008;29:2909-2945
      CrossRef | Web of Science | Medline

    1. 8Hochman JS, Sleeper LA, Webb JG, et al. Early revascularization in acute myocardial infarction complicated by cardiogenic shock. N Engl J Med 1999;341:625-634
      Full Text | Web of Science | Medline

    1. 9Binanay C, Califf RM, Hasselblad V, et al. Evaluation study of congestive heart failure and pulmonary artery catheterization effectiveness: the ESCAPE trial. JAMA 2005;294:1625-1633
      CrossRef | Web of Science | Medline

    1. 10Velazquez EJ, Lee KL, Deja MA, et al. Coronary-artery bypass surgery in patients with left ventricular dysfunction. N Engl J Med 2011;364:1607-1616
      Full Text | Web of Science | Medline

  1. 11Jollis JG, Roettig ML, Aluko AO, et al. Implementation of a statewide system for coronary reperfusion for ST-segment elevation myocardial infarction. JAMA 2007;298:2371-2380
    CrossRef | Web of Science | Medline

Related posts to this topic on this Scientific Web Site:

Foreseen changes in Guideline of Treatment of Cardiogenic Shock with Intra-aortic Balloon counterPulsation (IABP)

http://pharmaceuticalintelligence.com/2012/08/27/foreseen-changes-in-guideline-of-treatment-of-cardiogenic-shock-with-intra-aortic-balloon-counterpulsation-iabp/

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Reporter: Aviva Lev-Ari, PhD, RN

 

From the European Society of Cardiology meeting, today’s exclusive video library presents Sunday’s meeting highlights with Deepak Bhatt, MD and Anthony DeMaria, MD; hallway buzz on Prasugrel; expert commentaries with Dr. Bhatt on the PURE and Aldo-DHF trials; and the Hot Line I full length press conference.

 

‘On The Scene’ Video Library at ESC Congress 2012 – Day One

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Reporter and Curator: Aviva Lev-Ari, PhD, RN

WOEST (What is the Optimal Antiplatelet and Anticoagulant Therapy in Patients with Oral Anticoagulantion and Coronary Stenting): Get Rid Of The Aspirin In Triple Therapy

According to current guidelines and clinical practice, PCI patients already taking an oral anticoagulant generally end up on triple therapy comprising the anticoagulant plus clopidogrel and aspirin. However, there is no supporting evidence base for this approach and the triple therapy regimen is known to increase bleeding complications. Now a new study– the first randomized trial to address this situation, according to the investigators–  may have a large impact on clinical practice by demonstrating that the omission of aspirin in this context appears to be safe and may reduce adverse events.

Results of the WOEST (What is the Optimal Antiplatelet and Anticoagulant Therapy in Patients with Oral Anticoagulantion and Coronary Stenting) trial were presented by Willem Dewilde at the ESC in Munich today. Investigators in the Netherlands and Belgium randomized 573 patients to triple therapy or dual therapy of an anticoagulant plus clopidogrel for at least one month after implantation of a bare-metal stent or one year after a drug-eluting stent. Two-thirds of the patients were receiving oral anticoagulation for atrial fibrillation.

The primary endpoint, the total number of bleeding events, was dramatically reduced in the dual therapy group at one year:

  • 44.9% in the triple therapy group versus 19.5% (HR 0.36, CI 0.26-0.50)

There were 3 intracranial bleeds in each group. Most of the difference in bleeding occurred in TIMI minor and minimal bleeding. The difference in TIMI major bleeding (3.3% versus 5.8%) did not achieve statistical significance.

Clinical events, the trials’s secondary endpoint, were numerically lower in the dual therapy group. The difference in mortality achieved statistical significance.

  • Mortality: 7 deaths (2.6%) in the dual therapy group versus 18 deaths (6.4%) in the triple therapy group, p=0.027
  • MI: 3.3% versus 4.7%, p=0.382
  • TVR: 7.3% versus 6.8%, p=0.876
  • Stroke: 1.1% versus 2.9%, p=0.128)
  • Stent thrombosis: 1.5% versus 3.2%, p=0.165

“The WOEST study demonstrates that omitting aspirin leads to less bleedings but does not increase the risk of stent thrombosis, stroke or myocardial infarction,” said Dewilde in an ESC press release. “Although the number of patients in the trial is limited, this is an important finding with implications for future treatment and guidelines in this group of patients known to be at high risk of bleeding and thrombotic complications.”

David Holmes said the trial addressed “an incredibly important issue” and predicted that it would “change the way we practice medicine, it will change practice right away.” Keith Fox said that the evidence base prior to WOEST was extremely limited and that the trial showed that there was no hazard in doing without aspirin. The ESC discussant, Marco Valgimigli, said the trial showed it was safe to drop aspirin and provided another demonstration that “we have hit the wall” with anticoagulation.

Republished with permission from CardioExchange, a NEJM group publication.

http://www.forbes.com/sites/larryhusten/2012/08/28/woest-get-rid-of-the-aspirin-in-triple-therapy/

European Society of Cardiology: Prasugrel Can’t Top Clopidogrel in ACS

 By Todd Neale, Senior Staff Writer, MedPage Today

Published: August 26, 2012

Reviewed by Robert Jasmer, MD; Associate Clinical Professor of Medicine, University of California, San Francisco

MUNICH — For patients with unstable angina or non-ST-segment elevation myocardial infarction (non-STEMI) who do not undergo revascularization, increasing platelet inhibition may not improve outcomes, a randomized trial showed.

Added to a background of low-dose aspirin, prasugrel (Effient) did not significantly reduce the rate of MI, stroke, or cardiovascular death compared with clopidogrel (13.9% versus 16%, HR 0.91, 95% CI 0.79 to 1.05), according to Matthew Roe, MD, of Duke University in Durham, N.C.

The risk of severe bleeding was similar with both drugs, although minor and moderate bleeding were increased with prasugrel, Roe reported at the European Society of Cardiology meeting here. The findings were published simultaneously online in the New England Journal of Medicine.

“I think the outcome is a bit surprising because we think usually that more aggressive antiplatelet therapy, conceivably, in the face of an acute coronary syndrome and non-ST-elevation would lead to lesser adverse outcome from acute myocardial infarction or death,” said William Zoghbi, MD, from Methodist DeBakey Heart Center in Houston and president of the American College of Cardiology.

But he said clinicians need to respect the data “and start thinking about pathogenesis and what we’re trying to do with any of our new interventions.”

In patients with unstable angina or non-STEMI, practice guidelines call for angiography within 48 to 72 hours with provisional revascularization. Many of these patients do not ultimately undergo revascularization, placing them at greater risk compared with those who have their arteries opened with percutaneous coronary intervention (PCI) or coronary artery bypass graft (CABG).

Recommended medical therapy is with clopidogrel and aspirin, which is an approach that will not change from the current findings, Zoghbi said.

The purpose of the TRILOGY ACS trial was to explore whether using a more powerful platelet inhibitor — prasugrel — would improve outcomes compared clopidogrel (Plavix) in this high-risk patient subset.

The primary analysis involved 7,243 patients younger than 75 (mean age 62) who were receiving aspirin and were randomized to prasugrel 10 mg daily (or 5 mg daily for those weighing less than 132 pounds) or to clopidogrel 75 mg daily. The researchers recommended a daily aspirin dose of 100 mg or less.

A secondary, exploratory analysis involved 2,083 patients, 75 or older, who were randomized to prasugrel 5 mg daily or to clopidogrel 75 mg daily.

The lack of efficacy seen in the primary analysis of patients younger than 75 remained when patients of all ages were combined. There were no between-group differences for any of the components of the primary endpoint.

A prespecified secondary analysis taking multiple recurrent ischemic events into consideration showed a lower risk of MI, stroke, and cardiovascular death with prasugrel in the younger patients (HR 0.85, 95% CI 0.72 to 1.00, P=0.04), a finding consistent with the main results of the TRITON-TIMI 38 trial, which involved patients treated with PCI. The apparent benefit appeared after 12 months of treatment.

“Although this observation is exploratory, it raises the question of whether investigation of the multiplicity of ischemic events is warranted in future secondary-prevention trials, rather than solely analyzing the time to the first event, as has been traditional in studies involving patients who have had an acute coronary event,” the researchers wrote.

Rates of GUSTO severe or life threatening bleeding and TIMI major bleeding — as well as intracranial hemorrhage — were similar in the two groups in both the younger patients and in the overall study population. When minor and moderate bleeding events were added, the bleeding rate was higher with prasugrel.

There were no widespread differences between the groups in rates of nonhemorrhagic serious adverse events, but heart failure was more frequent with clopidogrel (1.8% versus 1.3%, P=0.045).

Douglas Weaver, MD, of Henry Ford Health System, said that he does not think the findings will have any impact on the use of prasugrel, which is not indicated for the patient population included in the study.

“It just doesn’t pass muster in improving value over clopidogrel,” said Weaver, a past president of the American College of Cardiology.

From a clinical perspective, he said, an important message from the study is the evidence of the safety of a reduced dose of prasugrel in the patients 75 and older, which is a consideration when prescribing prasugrel for patients undergoing PCI.

In comments following Roe’s presentation, Raffaele De Caterina, MD, PhD, of the G. d’Annunzio University in Chieti, Italy, provided context about how the findings fit in with the rest of the literature.

He compared the current results to those of a substudy of the PLATO trial, which involved ticagrelor (Brilinta).

In that trial, ticagrelor significantly reduced vascular death, MI, and stroke (HR 0.85, 95% CI 0.73 to 1.00, P=0.045) — the primary endpoint — and all-cause death (HR 0.75, 95% CI 0.61 to 0.93).

He then highlighted the ESC guidelines on treating patients with acute coronary syndromes without persistent ST-segment elevation.

In those, ticagrelor is recommended for all patients at moderate-to-high risk of ischemic events, regardless of initial treatment strategy and including those pre-treated with clopidogrel, and prasugrel is recommended for those who have not taken another P2Y12 inhibitor, who have a known coronary anatomy, and who are proceeding to PCI.

“I believe such statements and recommendations of the guidelines should not be changed,” De Caterina said.

TRILOGY ACS was funded by Eli Lilly and Daiichi Sankyo.

Roe reported relationships with Daiichi Sankyo, Eli Lilly, AstraZeneca, Bristol-Myers Squibb, Janssen Pharmaceuticals, Merck, Hoffmann-La Roche, and sanofi-aventis. The other authors reported numerous relationships with industry.

Primary source: New England Journal of Medicine

Prematurely halted ALTITUDE trial showed When added to monotherapy with either an ACE inhibitor or an angiotensin receptor blocker (ARB), aliskiren (Tekturna) did not improve outcomes in patients with type 2 diabetes who had high cardiovascular and renal risk

ESC: Aliskiren Onboard No Help in T2D

By Todd Neale, Senior Staff Writer, MedPage Today

Published: August 26, 2012

Reviewed by Robert Jasmer, MD; Associate Clinical Professor of Medicine, University of California, San Francisco

MUNICH — When added to monotherapy with either an ACE inhibitor or an angiotensin receptor blocker (ARB), aliskiren (Tekturna) did not improve outcomes in patients with type 2 diabetes who had high cardiovascular and renal risk, the prematurely halted ALTITUDE trial showed.

Through an average follow-up of 32 months, a composite of various cardiovascular and renal outcomes occurred in 17.9% of patients receiving the direct renin inhibitor and 16.8% of those receiving placebo (HR 1.08, 95% CI 0.98 to 1.20), according to Hans-Henrik Parving, MD, DMSc, of the University of Copenhagen and Aarhus University in Denmark.

As a Hot Line presentation European Society of Cardiology meeting here, Parving reported that there were no significant differences on any of the individual components of the endpoint — cardiovascular death, resuscitated sudden death, MI, stroke, unplanned hospitalization for heart failure, doubling of baseline serum creatinine, and onset of end-stage renal disease — or all-cause death.

The rate of stroke — mostly ischemic stroke — was numerically higher with aliskiren, although the result fell short of statistical significance (3.4% versus 2.8%; HR 1.25, 95% CI 0.98 to 1.60,P=0.07).

Thus, Parving said, using aliskiren with ACE inhibitors or ARBs in these high-risk patients “is not recommended and may even be harmful.”

The data monitoring committee for the ALTITUDE trial decided to stop the study early in December 2011 both for futility and for adverse events. Then, earlier this year, the FDA issued a warning about using aliskiren with ACE inhibitors or ARBs and changed the drug label to reflect a contraindication for such combinations in patients with diabetes or renal impairment.

The trial included 8,561 patients with type 2 diabetes who had a high risk of cardiovascular or renal disease who were randomized to aliskiren — at 150 mg daily for 1 month followed by 300 mg daily thereafter — or placebo in addition to monotherapy with either an ACE inhibitor or an ARB (but not both).

Adding aliskiren did not improve outcomes, and in fact, may have caused harm, Parving said, as indicated by the apparent increase in stroke risk.

He said that could be explained by the impaired autoregulation of patients with diabetes or by chance, as there are no indications of a stroke risk in other studies of the drug.

Johannes Mann, of Friedrich Alexander University in Erlangen, Germany, and McMaster University in Hamilton, Ontario, who served as the discussant following Parving’s presentation, agreed that it could be a chance finding, but said that it could also be a direct effect of aliskiren itself.

He concluded that the stroke risk was not explained, however, by dual renin system inhibition, because such a signal was not seen in the ONTARGET trial, which compared the combination of ramipril (an ACE inhibitor) and telmisartan (an ARB) with each drug as monotherapy.

As noted when the trial was halted last year, adverse events were more frequent in the aliskiren group.

The percentage of patients who had a potassium level of 5.5 to less than 6.0 mmol/L was greater with active treatment (21% versus 16%), as was the percentage of those with a potassium level of 6.0 mmol/L or greater (8.8% versus 5.6%).

Aliskiren carried higher risks of hyperkalemia (38.7% versus 28.6%), hypotension (12.1% versus 8%), diarrhea (9.6% versus 7.2%), and falls (2.8% versus 2.6%). There was one death caused by hyperkalemia.

Douglas Weaver, MD, of the Henry Ford Health System in Detroit, said that the findings were disappointing, but that they likely wouldn’t change how aliskiren is used in practice.

“I don’t think this is going to have a negative or a positive effect on it,” said Weaver, who is a past president of the American College of Cardiology.

ALTITUDE was sponsored by Novartis Pharma AG.

The executive committee and other investigators or their institutions received a consultancy fee. Some of the authors are employees of Novartis and therefore eligible for stock and stock options.

Primary source: European Society of Cardiology
Source reference:
Parving H-H, et al “The Aliskiren Trial in Type 2 Diabetes Using Cardio-Renal Endpoints (ALTITUDE)” ESC 2012; Abstract 399.

Aliskiren

From Wikipedia, the free encyclopedia
Aliskiren
Systematic (IUPAC) name
(2S,4S,5S,7S)-5-amino-N-(2-carbamoyl-2,2-dimethylethyl)-4-hydroxy-7-{[4-methoxy-3-(3-methoxypropoxy)phenyl]methyl}-8-methyl-2-(propan-2-yl)nonanamide
Aliskiren (INN) (trade names Tekturna, U.S.; Rasilez, U.K. and elsewhere) is the first in a class of drugs called direct renin inhibitors. Its current licensed indication is essential (primary) hypertension.

Aliskiren was co-developed by the Swiss pharmaceutical companies Novartis and Speedel.[1][2] It was approved by the U.S. Food and Drug Administration in 2007 for the treatment of primary hypertension.[3]

In December 2011, Novartis had to halt a clinical trial of the drug after discovering increased incidence of non-fatal stroke, renal complications, hyperkalemia and hypotension in patients with diabetes and renal impairment.[4]

The following recommendations are being added to the drug labels for aliskiren-containing products as of 4/20/12:

I) A new contraindication against the use of aliskiren with ARBs or ACEIs in patients with diabetes because of the risk of renal impairment, hypotension, and hyperkalemia. II) A warning to avoid use of aliskiren with ARBs or ACEIs in patients with moderate to severe renal impairment (i.e., where glomerular filtration rate [GFR] < 60 mL/min).

Mechanism of Action

Renin is the first enzyme in the renin-angiotensin-aldosterone system which plays a role in blood pressure control. Renin cleaves angiotensinogen to angiotensin I, which is in turn converted by angiotensin-converting enzyme (ACE) toangiotensin II. Angiotensin II has both direct and indirect effects on blood pressure. It directly causes arterial smooth muscle to contract, leading to vasoconstriction and increased blood pressure. Angiotensin II also stimulates the production of aldosterone from the adrenal cortex, which causes the tubules of the kidneys to increase reabsorption of sodium, with water following thereby increasing plasma volume and blood pressure.

Aliskiren binds to the S3bp binding pocket of renin, essential for its activity.[5] Binding to this pocket prevents the conversion of angiotensinogen to angiotensin I.
Aliskiren is also available as combination therapy with hydrochlorothiazide.[6]

Many drugs control blood pressure by interfering with angiotensin or aldosterone. However, when these drugs are used chronically, the body increases renin production, which drives blood pressure up again. Therefore, doctors have been looking for a drug to inhibit renin directly. Aliskiren is the first drug to do so.[7][8]

Aliskiren may have renoprotective effects that are independent of its blood pressure−lowering effect in patients with hypertension, type 2 diabetes, and nephropathy who are receiving the recommended renoprotective treatment. According to the AVOID study, researchers found that treatment with 300 mg of aliskiren daily, as compared with placebo, reduced the mean urinary albumin-to-creatinine ratio by 20% (95% confidence interval, 9 to 30; P<0.001), with a reduction of 50% or more in 24.7% of the patients who received aliskiren as compared with 12.5% of those who received placebo (P<0.001). Furthermore, the AVOID trial shows that treatment with 300 mg of aliskiren daily reduces albuminuria in patients with hypertension, type 2 diabetes, and proteinuria who are receiving the recommended maximal renoprotective treatment with losartan and optimal antihypertensive therapy. Therefore, direct renin inhibition will have a critical role in strategic renoprotective pharmacotherapy, in conjunction with dual blockade of the renin−angiotensin−aldosterone system with the use of ACE inhibitors and angiotensin II–receptor blockers, very high doses of angiotensin II−receptor blockers, and aldosterone blockade.[9]

Adverse effects

  • Angioedema
  • Hyperkalemia (particularly when used with ACE inhibitors in diabetic patients)
  • Hypotension (particularly in volume-depleted patients)
  • Diarrhea and other GI symptoms
  • Headache
  • Dizziness
  • Cough
  • Rash
  • Elevated uric acidgout, and renal stones
  • Rarely: allergic swelling of the face, lips or tongue and difficulty breathing

Contraindications

  • Pregnancy: other drugs such as ACE inhibitors, also acting on the renin-angiotensin system have been associated with fetal malformations and neonatal death[10]
  • Breast feeding: during animal studies, the drug has been found present in milk.[10]

Aliskiren has not yet been evaluated in patients with significantly impaired renal function.

Drug interactions

Aliskiren is a minor substrate of CYP3A4 and, more important, P-glycoprotein:

  • Reduces furosemide blood concentration.
  • Atorvastatin may increase blood concentration, however no dose adjustment needed.
  • Possible interaction with ciclosporin (the concomitant use of ciclosporin and aliskiren is contraindicated).
  • Caution should be exercised when aliskiren is administered with ketoconazole or other moderate P-gp inhibitors (itraconazole, clarithromycin, telithromycin, erythromycin, amiodarone).
  • Doctors should stop prescribing aliskiren-containing medicines to patients with diabetes (type 1 or type 2) or with moderate to severe kidney impairment who are also taking an ACE inhibitor or ARB, and should consider alternative antihypertensive treatment as necessary.[11]

References

  1. ^ Gradman A, Schmieder R, Lins R, Nussberger J, Chiang Y, Bedigian M (2005). “Aliskiren, a novel orally effective renin inhibitor, provides dose-dependent antihypertensive efficacy and placebo-like tolerability in hypertensive patients”. Circulation 111 (8): 1012–8. doi:10.1161/01.CIR.0000156466.02908.EDPMID 15723979.
  2. ^ Straessen JA, Li Y, and Richart T (2006). “Oral Renin Inhibitors”Lancet 368 (9545): 1449–56. doi:10.1016/S0140-6736(06)69442-7PMID 17055947.
  3. ^ “First Hypertension Drug to Inhibit Kidney Enzyme Approved”CBC. 2007-03-06. Retrieved 2007-03-14.[dead link]
  4. ^ Healthzone.ca: Blood-pressure drug reviewed amid dangerous side effects
  5. ^ “Chemistry & Biology : Structure-based drug design: the discovery of novel nonpeptide orally active inhibitors of human renin”. ScienceDirect. Retrieved 2010-01-20.
  6. ^ Baldwin CM, Plosker GL.[1]doi:10.2165/00003495-200969070-00004. Drugs 2009; 69(7):833-841.
  7. ^ Ingelfinger JR (June 2008). “Aliskiren and dual therapy in type 2 diabetes mellitus”N. Engl. J. Med. 358 (23): 2503–5. doi:10.1056/NEJMe0803375.PMID 18525047.
  8. ^ PharmaXChange: Direct Renin Inhibitors as Antihypertensive Drugs
  9. ^ Parving HH, Persson F, Lewis JB, Lewis EJ, Hollenberg NK. “Aliskiren Combined with Losartan in Type 2 Diabetes and Nephropathy,” N Engl J Med 2008;358:2433-46.
  10. a b Drugs.com: Tekturna
  11. ^ European Medicines Agency recommends new contraindications and warnings for aliskiren-containing medicines.

External links

 http://en.wikipedia.org/wiki/Aliskiren

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