The value of national institutes of health (NIH) registry-based research in identifying childhood cardiac disease outcomes: The pediatric cardiomyopathy registry experience

Cardiomyopathy is a serious, but rare, disease of the heart muscle. Cardiomyopathy commonly results in heart failure and is the leading cause of heart transplantation in children older than 1 year of age. The Pediatric Cardiomyopathy Registry (PCMR), which has been funded by the National Heart Lung and Blood Institute (NHBLI) since 1994, has enrolled and followed more than 3,500 children with cardiomyopathy in the US and Canada. Results from the registry have established the incidence of pediatric cardiomyopathy in North America, determined the prevalence of heart failure, have identified causes of cardiomyopathy, as well as transplant and survival patterns. In this chapter, we will describe the design and operation of the PCMR and present a summary of results. The PCMR is an example of how a well-designed and well-conducted patient registry can provide important insights into the etiologies, clinical course, and patient outcomes for a rare disease, in this case cardiomyopathy in children.