Idiopathic and Primary Cardiomyopathy in Children: Research Directions and Strategies Conference

Bethesda, MD
January 25-26, 2007

Chair of the Conference
Steven E. Lipshultz, M.D.
Leonard M. Miller School of Medicine,
University of Miami

Co-Chairs of the Conference
Steven Colan, M.D.
Children's Hospital Boston
Harvard Medical School

Jeffrey A. Towbin, M.D.
Texas Children's Hospital
Baylor College of Medicine

James D. Wilkinson, M.D., M.P.H.
Leonard M. Miller School of Medicine,
University of Miami
Cardiomyopathies result in some of the worst pediatric cardiology outcomes, as nearly as 40% of the children who present with symptomatic 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 have shown that etiologies are established in very few children with cardiomyopathy 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 are much less than for these other conditions.

In January 2007, the first scientific conference on future research directions for children with idiopathic or primary cardiomyopathies was held in Bethesda, Maryland. The conference was sponsored by the Children's Cardiomyopathy Foundation and the National Heart, Lung and Blood Institute with additional support from the Genzyme Corporation and the Department of Pediatrics of the Miller School of Medicine at the University of Miami. Leading basic and clinical investigators in pediatric cardiomyopathy from across the United States, Canada, and Australia participated in the conference activities.

The aims of the workshop were 1) to review the current understanding of the molecular and genetic issues in cardiomyopathy; the epidemiology, etiologies and outcomes of cardiomyopathy; and clinical issues including medical management, cardiac transplantation and health related quality of life; 2) to identify the most critical and promising areas for future basic science and clinical research efforts regarding pediatric cardiomyopathy and 3) to disseminate the results and recommendations of the workshop.

Conference Presentations

Thursday, January 25, 2007

Opening Remarks

Workshop 1: Molecular and Genetic Issues

Workshop 2: Epidemiology, Etiology, and Outcomes


Friday, January 26, 2007

Young Investigator Presentations

Workshop 3: Clinical Issues in Pediatric Cardiomyopathy

Closing Remarks