Primary Cardiomyopathies in Children: Research Directions & Strategies

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DESCRIPTION (provided by applicant): Cardiomyopathy is a serious disease of the heart muscle. Nearly 40 percent of children with cardiomyopathy receive a heart transplant or die within the first 2 years after diagnosis. The percentage of children with cardiomyopathy who received a heart transplant has not declined over the past 10 years and cardiomyopathy remains the leading cause of transplantation for children over one year of age. Studies from the NHLBI-funded Pediatric Cardiomyopathy Registry (PCMR) have shown that the cause of cardiomyopathy is established in very few children yet genetic causes are likely to be present in most. The incidence of pediatric cardiomyopathy is approximately 1 per 100,000 children. This is comparable to the incidence of such childhood cancers such as lymphoma, Wilms' tumor, and neuroblastoma. However, the published research and scientific conferences focused on pediatric cardiomyopathy is much less than for these other conditions. The purpose of the proposed scientific workshop, "Idiopathic and Primary Cardiomyopathy in Children, will be to review the current state/knowledge of genetic and clinical research on cardiomyopathy to determine what new molecular studies, genetic diagnostic tests or therapeutic studies should be initiated and developed to improve detection and treatment in cases of cardiomyopathy in children without a known cause. This meeting is designed to bring together a multidisciplinary group of scientists, including molecular biologists, geneticists, epidemiologists and clinicians in order to share the most up to date research on the prevention, cause, diagnosis, and treatment of PCM and shape the direction of future research on the disease. It is anticipated that this exchange of knowledge will identify future research directions and new approaches and technologies that will lead to a better understanding of this disease and improved treatment and prevention strategies.