title: The pediatric cardiomyopathy registry and heart failure: key results from the first 15 years.
abstract: Cardiomyopathy is a serious disorder of the heart muscle and, although rare, is a common cause of heart failure in children and the most common cause for heart transplantation in children older than 1 year of age. Funded by the National Heart Lung and Blood Institute since 1994, the Pediatric Cardiomyopathy Registry (PCMR) has followed more than 3500 North American children with cardiomyopathy. Early analyses determined estimates for the incidence of pediatric cardiomyopathy (1.13 cases per 100,000 children per year), risk factors for cardiomyopathy (age <1 year, male sex, black race, and living in New England as opposed to the central southwestern states), the prevalence of heart failure at diagnosis (6%-84% depending on cause), and 10-year survival (29%-94% depending on cause). More recent analyses explored cause-specific functional status, survival and transplant outcomes, and risk factors in greater detail. For many topics these analyses are based on the largest and best-documented samples of children with disease such as the muscular dystrophies, mitochondrial disorders, and Noonan syndrome. Data from the PCMR continue to provide valuable information that guides clinical management and the use of life-saving therapies, such as cardiac transplantation and approaches to treating heart failure, and prepares children, their families, and their caregivers to deal with this serious condition.
authors.affiliation: Department of Pediatrics D820, Leonard M Miller School of Medicine, University of Miami, PO Box 016820, Miami, FL 33101, USA.
authors.forename: James D
authors.initials: JD
authors.lastname: Wilkinson
authors.forename: David C
authors.initials: DC
authors.lastname: Landy
authors.forename: Steven D
authors.initials: SD
authors.lastname: Colan
authors.forename: Jeffrey A
authors.initials: JA
authors.lastname: Towbin
authors.forename: Lynn A
authors.initials: LA
authors.lastname: Sleeper
authors.forename: E John
authors.initials: EJ
authors.lastname: Orav
authors.forename: Gerald F
authors.initials: GF
authors.lastname: Cox
authors.forename: Charles E
authors.initials: CE
authors.lastname: Canter
authors.forename: Daphne T
authors.initials: DT
authors.lastname: Hsu
authors.forename: Steven A
authors.initials: SA
authors.lastname: Webber
authors.forename: Steven E
authors.initials: SE
authors.lastname: Lipshultz
mesh_terms: D002311:Cardiomyopathy, Dilated / Q000453:epidemiology* / Q000209:etiology / Q000628:therapy*; D002312:Cardiomyopathy, Hypertrophic / Q000453:epidemiology* / Q000209:etiology / Q000628:therapy*; D002316:Cardiotonic Agents / Q000627:therapeutic use; D002648:Child; D003226:Congresses as Topic; D006130:Growth Disorders / Q000209:etiology; D006333:Heart Failure / Q000453:epidemiology / Q000209:etiology / Q000628:therapy; D016027:Heart Transplantation; D006801:Humans; D015994:Incidence; D054555:National Heart, Lung, and Blood Institute (U.S.); D015995:Prevalence; D012042:Registries; D012307:Risk Factors; D014481:United States / Q000453:epidemiology
chemical_list: D002316:Cardiotonic Agents