Epidemiological and outcomes research in children with pediatric cardiomyopathy: Discussions from the international workshop on primary and idiopathic cardiomyopathies in children

Abstract 

This report summarizes the roundtable discussion held at the first International Workshop on Primary and Idiopathic Cardiomyopathies in Children which focused on future directions for research on the epidemiology, etiology and outcomes for children with cardiomyopathy. Areas identified as important for future research included: 1) developing a standardized approach to the assessment and follow-up of children with myocarditis; 2) investigating the epidemiology of sudden death in children with dilated cardiomyopathy; 3) identification of biomarkers to serve as surrogate endpoints for important clinical outcomes; 4) the continuation of observational studies like the National Heart, Lung, and Blood Institute-sponsored Pediatric Cardiomyopathy Registry; and 5) conducting randomized clinical trials of pharmacological and behavioral interventions. It was concluded that optimal research strategies should employ a multidisciplinary research team including pediatric cardiologists, epidemiologists, biostatisticians, geneticists, patient care staff and advocacy groups. Further, adequately powered clinical trials may be facilitated by the establishment of a pediatric cardiomyopathy clinical trials network.

Keywords: Cardiomyopathy, Epidemiology, Sudden death, Myocarditis, Research