JAMESFETT
08-22-2008, 09:15 AM
An important proposal for comprehensive study of PPCM in 12 major USA medical centers has been submitted, Let us hope and pray that funding will be forthcoming so that the much-needed study can begin. This brings together all the important body of knowledge that has accumulated since 2000 about PPCM. There is definitely progress. Kudos also to AMH.org (see P.S.)
PROGRAM INTRODUCTION AND STATEMENT OF OBJECTIVES
Peripartum Cardiomyopathy and Myocarditis Consortium (PCMC): This Rare Disease Clinical Research Network (RDCRN) proposal will establish a Peripartum Cardiomyopathy and Myocarditis Consortium (PCMC) to advance clinical research and improve clinical care for two important but rare disorders which initiate the pathogenesis of non-ischemic cardiomyopathy: peripartum cardiomyopathy (PPCM) and inflammatory myocarditis. Though theoretically initiated by different factors, maternal-fetal interactions in PPCM (Pearson, JAMA, 2000) and viral infections in myocarditis (Feldman et al. NEJM, 2000), these disorders share a common immune pathogenesis which results in myocardial injury and left ventricular dysfunction. Dramatic myocardial recovery is frequently evident in both PPCM and myocarditis; however, those subjects who do not recover are left with dilated cardiomyopathy (DCM), a disease which progresses over time and leads to cardiac
transplantation or death. Research into the pathogenesis of both disorders has been limited by their infrequent occurrence at most individual referral centers. In addition, the inability to predict which subjects will recover makes clinical trials of therapeutic innovation difficult. The PCMC will bring together 12 major centers of clinical care and research in heart failure to further understanding of pathogenesis and optimize and improve current standards of clinical care. In addition through
investigation of novel transcriptome-based biomarkers (TBB), the network proposal seeks to improve our ability to predict recovery and set the stage for future interventional trials in those subjects who will not recover with current therapeutics.
Aim 1: Establish a multi-center Peripartum Cardiomyopathy and Myocarditis Consortium (PCMC) to foster and facilitate clinical investigation in peripartum cardiomyopathy and acute myocarditis. The network incorporates twelve major U.S. heart failure clinical research centers (University of Pittsburgh, Mayo Clinic, Cleveland Clinic, Massachusetts General Hospital, Johns Hopkins, University of Rochester, University of Texas Southwestern, Penn State Medical Center, Thomas Jefferson Medical College, University of Miami, Baylor Children’s and UCLA), and will combine their clinical and research expertise with the resources of Clinical Translational Science Institutes at seven sites to form the core of the consortium. The PCMC would serve as the focal point for research in both acute peripartum cardiomyopathy and the related disorders of acute inflammatory myocarditis. To achieve this aim, the following activities are proposed: A) Develop a clinical data
repository in collaboration with other Rare Disease Clinical Research Consortia and the Rare Disease Data and Technology Coordinating Center. B) Build a clinical specimen bank for storage of serum, plasma, DNA, and tissue samples linked to the clinical data repository. C) Enact a national recruitment program in cooperation with patient advocacy groups. D) Utilize the extensive resources of the Clinical Translational Science Institutes at seven of the consortium sites.
Aim 2: Conduct longitudinal studies of the immune etiology of PPCM and of viral etiologies of acute myocarditis and their relationship to myocardial recovery and clinical outcomes. These longitudinal investigations will utilize the multi-center network to further the understanding of their pathogenesis, and will develop novel biomarkers of inflammatory disease activity. Utilizing the consortium Clinical Data Repository and Clinical Specimen Bank and in coordination with biostatistical support from the Data and Technology Coordinating Center, the Consortium Biomarkers Project will use several promising techniques to develop new markers of disease activity. This program will establish a framework for testing of future novel biomarkers by investigators both within and beyond the consortium.
JD
P.S. The proposal also identifies AMH.org as participants in this project, as follows:
Collaborative Arrangements with Patient Support Organizations:
The PCMC will work together with important patient advocacy groups, “A Mothers Heart (PPCM, Ms. Serena Welsh, CEO, Dr. James Fett, medical advisor) and the Myocarditis Foundation (myocarditis, Ms. Candace Moose, Secretary and Treasurer, Dr Leslie Cooper, Medical Director. Both advocacy groups have expressed their support for the proposed network and their willingness to work together toward mutual goals. Both “A Mothers Heart” and the “Myocarditis Foundation” maintain active web sites which each receive several million “hits” each year. These web sites will be “linked” in a bidirectional manner to allow patients hitting the web sites to gain access to the consortium web sites and vice versa. In addition, a consistent concern among patients with these rare disorders is the lack of physician awareness as to the appropriate diagnostic workup, therapy and outcomes. Educating physicians about the state of the art of diagnosis and treatment of PPCM and myocarditis will be an important mutual goal of the consortium. An education committee made up from the leadership of patient advocacy groups and the leadership of the consortium will be established to review education goals and materials. The leadership of the consortium will work closely with the leadership of “A Mothers Heart” and “The Myocarditis Foundation” to improve access to care, education of subjects with these disorders and their physicians, and to develop web based tools for bidirectional communication.
PROGRAM INTRODUCTION AND STATEMENT OF OBJECTIVES
Peripartum Cardiomyopathy and Myocarditis Consortium (PCMC): This Rare Disease Clinical Research Network (RDCRN) proposal will establish a Peripartum Cardiomyopathy and Myocarditis Consortium (PCMC) to advance clinical research and improve clinical care for two important but rare disorders which initiate the pathogenesis of non-ischemic cardiomyopathy: peripartum cardiomyopathy (PPCM) and inflammatory myocarditis. Though theoretically initiated by different factors, maternal-fetal interactions in PPCM (Pearson, JAMA, 2000) and viral infections in myocarditis (Feldman et al. NEJM, 2000), these disorders share a common immune pathogenesis which results in myocardial injury and left ventricular dysfunction. Dramatic myocardial recovery is frequently evident in both PPCM and myocarditis; however, those subjects who do not recover are left with dilated cardiomyopathy (DCM), a disease which progresses over time and leads to cardiac
transplantation or death. Research into the pathogenesis of both disorders has been limited by their infrequent occurrence at most individual referral centers. In addition, the inability to predict which subjects will recover makes clinical trials of therapeutic innovation difficult. The PCMC will bring together 12 major centers of clinical care and research in heart failure to further understanding of pathogenesis and optimize and improve current standards of clinical care. In addition through
investigation of novel transcriptome-based biomarkers (TBB), the network proposal seeks to improve our ability to predict recovery and set the stage for future interventional trials in those subjects who will not recover with current therapeutics.
Aim 1: Establish a multi-center Peripartum Cardiomyopathy and Myocarditis Consortium (PCMC) to foster and facilitate clinical investigation in peripartum cardiomyopathy and acute myocarditis. The network incorporates twelve major U.S. heart failure clinical research centers (University of Pittsburgh, Mayo Clinic, Cleveland Clinic, Massachusetts General Hospital, Johns Hopkins, University of Rochester, University of Texas Southwestern, Penn State Medical Center, Thomas Jefferson Medical College, University of Miami, Baylor Children’s and UCLA), and will combine their clinical and research expertise with the resources of Clinical Translational Science Institutes at seven sites to form the core of the consortium. The PCMC would serve as the focal point for research in both acute peripartum cardiomyopathy and the related disorders of acute inflammatory myocarditis. To achieve this aim, the following activities are proposed: A) Develop a clinical data
repository in collaboration with other Rare Disease Clinical Research Consortia and the Rare Disease Data and Technology Coordinating Center. B) Build a clinical specimen bank for storage of serum, plasma, DNA, and tissue samples linked to the clinical data repository. C) Enact a national recruitment program in cooperation with patient advocacy groups. D) Utilize the extensive resources of the Clinical Translational Science Institutes at seven of the consortium sites.
Aim 2: Conduct longitudinal studies of the immune etiology of PPCM and of viral etiologies of acute myocarditis and their relationship to myocardial recovery and clinical outcomes. These longitudinal investigations will utilize the multi-center network to further the understanding of their pathogenesis, and will develop novel biomarkers of inflammatory disease activity. Utilizing the consortium Clinical Data Repository and Clinical Specimen Bank and in coordination with biostatistical support from the Data and Technology Coordinating Center, the Consortium Biomarkers Project will use several promising techniques to develop new markers of disease activity. This program will establish a framework for testing of future novel biomarkers by investigators both within and beyond the consortium.
JD
P.S. The proposal also identifies AMH.org as participants in this project, as follows:
Collaborative Arrangements with Patient Support Organizations:
The PCMC will work together with important patient advocacy groups, “A Mothers Heart (PPCM, Ms. Serena Welsh, CEO, Dr. James Fett, medical advisor) and the Myocarditis Foundation (myocarditis, Ms. Candace Moose, Secretary and Treasurer, Dr Leslie Cooper, Medical Director. Both advocacy groups have expressed their support for the proposed network and their willingness to work together toward mutual goals. Both “A Mothers Heart” and the “Myocarditis Foundation” maintain active web sites which each receive several million “hits” each year. These web sites will be “linked” in a bidirectional manner to allow patients hitting the web sites to gain access to the consortium web sites and vice versa. In addition, a consistent concern among patients with these rare disorders is the lack of physician awareness as to the appropriate diagnostic workup, therapy and outcomes. Educating physicians about the state of the art of diagnosis and treatment of PPCM and myocarditis will be an important mutual goal of the consortium. An education committee made up from the leadership of patient advocacy groups and the leadership of the consortium will be established to review education goals and materials. The leadership of the consortium will work closely with the leadership of “A Mothers Heart” and “The Myocarditis Foundation” to improve access to care, education of subjects with these disorders and their physicians, and to develop web based tools for bidirectional communication.