title: Lessons learned from the Pediatric Cardiomyopathy Registry (PCMR) Study Group.
abstract: Cardiomyopathy is a rare disorder of the heart muscle, affecting 1.13 cases per 100,000 children, from birth to 18 years of age. Cardiomyopathy is the leading cause of heart transplantation in children over the age of 1. The Pediatric Cardiomyopathy Registry funded in 1994 by the National Heart, Lung, and Blood Institute was established to examine the epidemiology of the disease in children below 18 years of age. More than 3500 children across the United States and Canada have been enrolled in the Pediatric Cardiomyopathy Registry, which has followed-up these patients until death, heart transplantation, or loss to follow-up. The Pediatric Cardiomyopathy Registry has provided the most in-depth illustration of this disease regarding its aetiology, clinical course, associated risk factors, and patient outcomes. Data from the registry have helped in guiding the clinical management of cardiomyopathy in children under 18 years of age; however, questions still remain regarding the most clinically effective diagnostic and treatment approaches for these patients. Future directions of the registry include the use of next-generation whole-exome sequencing and cardiac biomarkers to identify aetiology-specific treatments and improve diagnostic strategies. This article provides a brief synopsis of the work carried out by the Pediatric Cardiomyopathy Registry since its inception, including the current knowledge on the aetiologies, outcomes, and treatments of cardiomyopathy in children.
authors.affiliation: 1Department of Pediatrics,Wayne State University School of Medicine,Children's Research Center of Michigan,Detroit,Michigan,United States of America.
authors.forename: James D
authors.initials: JD
authors.lastname: Wilkinson
authors.affiliation: 1Department of Pediatrics,Wayne State University School of Medicine,Children's Research Center of Michigan,Detroit,Michigan,United States of America.
authors.forename: Joslyn A
authors.initials: JA
authors.lastname: Westphal
authors.affiliation: 2Department of Pediatrics,Detroit Medical Center,Children's Hospital of Michigan,Detroit,Michigan,United States of America.
authors.forename: Neha
authors.initials: N
authors.lastname: Bansal
authors.affiliation: 1Department of Pediatrics,Wayne State University School of Medicine,Children's Research Center of Michigan,Detroit,Michigan,United States of America.
authors.forename: Jason D
authors.initials: JD
authors.lastname: Czachor
authors.affiliation: 1Department of Pediatrics,Wayne State University School of Medicine,Children's Research Center of Michigan,Detroit,Michigan,United States of America.
authors.forename: Hiedy
authors.initials: H
authors.lastname: Razoky
authors.affiliation: 1Department of Pediatrics,Wayne State University School of Medicine,Children's Research Center of Michigan,Detroit,Michigan,United States of America.
authors.forename: Steven E
authors.initials: SE
authors.lastname: Lipshultz
mesh_terms: D002170:Canada; D009202:Cardiomyopathies / Q000145:classification* / Q000150:complications* / Q000235:genetics*; D019468:Disease Management; D006333:Heart Failure / Q000453:epidemiology* / Q000628:therapy; D016027:Heart Transplantation / Q000379:methods; D006801:Humans; D010372:Pediatrics; D012042:Registries / Q000592:standards*; D012307:Risk Factors; D014481:United States
keywords: Cardiomyopathy; Pediatric Cardiomyopathy Registry; heart failure; paediatrics; patient registry