Pediatric Cardiomyopathies

Title

Pediatric Cardiomyopathies

Creator

Lee TM; Hsu DT; Kantor P; Towbin JA; Ware SM; Colan SD; Chung WK; Jefferies JL; Rossano JW; Castleberry CD; Addonizio LJ; Lal AK; Lamour JM; Miller EM; Thrush PT; Czachor JD; Razoky H; Hill A; Lipshultz SE

Identifier

10.1161/circresaha.116.309386

Publisher

Circulation Research

Date

2017

Subject

Epidemiology; Genetics; Pediatrics

Description

Pediatric cardiomyopathies are rare diseases with an annual incidence of 1.1 to 1.5 per 100 000. Dilated and hypertrophic cardiomyopathies are the most common; restrictive, noncompaction, and mixed cardiomyopathies occur infrequently; and arrhythmogenic right ventricular cardiomyopathy is rare. Pediatric cardiomyopathies can result from coronary artery abnormalities, tachyarrhythmias, exposure to infection or toxins, or secondary to other underlying disorders. Increasingly, the importance of genetic mutations in the pathogenesis of isolated or syndromic pediatric cardiomyopathies is becoming apparent. Pediatric cardiomyopathies often occur in the absence of comorbidities, such as atherosclerosis, hypertension, renal dysfunction, and diabetes mellitus; as a result, they offer insights into the primary pathogenesis of myocardial dysfunction. Large international registries have characterized the epidemiology, cause, and outcomes of pediatric cardiomyopathies. Although adult and pediatric cardiomyopathies have similar morphological and clinical manifestations, their outcomes differ significantly. Within 2 years of presentation, normalization of function occurs in 20% of children with dilated cardiomyopathy, and 40% die or undergo transplantation. Infants with hypertrophic cardiomyopathy have a 2-year mortality of 30%, whereas death is rare in older children. Sudden death is rare. Molecular evidence indicates that gene expression differs between adult and pediatric cardiomyopathies, suggesting that treatment response may differ as well. Clinical trials to support evidence-based treatments and the development of disease-specific therapies for pediatric cardiomyopathies are in their infancy. This compendium summarizes current knowledge of the genetic and molecular origins, clinical course, and outcomes of the most common phenotypic presentations of pediatric cardiomyopathies and highlights key areas where additional research is required. CLINICAL TRIAL REGISTRATION: URL: http://www.clinicaltrials.gov. Unique identifiers: NCT02549664 and NCT01912534.

Rights

Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).

Citation List Month

November 2017 List

Notes

1524-4571
Lee, Teresa M
Hsu, Daphne T
Kantor, Paul
Towbin, Jeffrey A
Ware, Stephanie M
Colan, Steven D
Chung, Wendy K
Jefferies, John L
Rossano, Joseph W
Castleberry, Chesney D
Addonizio, Linda J
Lal, Ashwin K
Lamour, Jacqueline M
Miller, Erin M
Thrush, Philip T
Czachor, Jason D
Razoky, Hiedy
Hill, Ashley
Lipshultz, Steven E
Journal Article
Review
United States
Circ Res. 2017 Sep 15;121(7):855-873. doi: 10.1161/CIRCRESAHA.116.309386.

Citation

Lee TM; Hsu DT; Kantor P; Towbin JA; Ware SM; Colan SD; Chung WK; Jefferies JL; Rossano JW; Castleberry CD; Addonizio LJ; Lal AK; Lamour JM; Miller EM; Thrush PT; Czachor JD; Razoky H; Hill A; Lipshultz SE, “Pediatric Cardiomyopathies,” Pediatric Palliative Care Library, accessed December 5, 2021, https://pedpalascnetlibrary.omeka.net/items/show/11019.

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