Cardiomyopathy Phenotypes and Outcomes for Children With Left Ventricular Myocardial Noncompaction: Results From the Pediatric Cardiomyopathy Registry.

Abstract

BACKGROUND:

Left ventricular noncompaction (LVNC) is a distinct form of cardiomyopathy characterized by hypertrabeculation of the left ventricle. The LVNC phenotype may occur in isolation or with other cardiomyopathy phenotypes. Prognosis is incompletely characterized in children.

METHODS AND RESULTS:

According to diagnoses from the National Heart, Lung, and Blood Institute-funded Pediatric Cardiomyopathy Registry from 1990 to 2008, 155 of 3,219 children (4.8%) had LVNC. Each LVNC patient was also classified as having an associated echocardiographically diagnosed cardiomyopathy phenotype: dilated (DCM), hypertrophic (HCM), restrictive (RCM), isolated, or indeterminate. The time to death or transplantation differed among the phenotypic groups (P = .035). Time to listing for cardiac transplantation significantly differed by phenotype (P < .001), as did time to transplantation (P = .015). The hazard ratio for death/transplantation (with isolated LVNC as the reference group) was 4.26 (95% confidence interval [CI] 0.78-23.3) for HCM, 6.35 (95% CI 1.52-26.6) for DCM, and 5.66 (95% CI 1.04-30.9) for the indeterminate phenotype. Most events occurred in the 1st year after diagnosis.

CONCLUSIONS:

LVNC is present in at least 5% of children with cardiomyopathy. The specific LVNC-associated cardiomyopathy phenotype predicts the risk of death or transplantation and should inform clinical management.

• Authors: John L Jefferies, James D Wilkinson, Lynn A Sleeper, Steven D Colan, Minmin Lu, Elfriede Pahl, Paul F Kantor, Melanie D Everitt, Steven A Webber, Beth D Kaufman, Jacqueline M Lamour, Charles E Canter, Daphne T Hsu, Linda J Addonizio, Steven E Lipshultz, Jeffrey A Towbin, Pediatric Cardiomyopathy Registry Investigators
• Journal: Journal of cardiac failure (J Card Fail) Vol. 21 Issue 11 Pg. 877-84 (Nov 2015) ISSN: 1532-8414 [Electronic] United States
• PMID: 26164213 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2015 Elsevier Inc. All rights reserved.

Topics

• Canada
• Cardiomyopathies (genetics, mortality, physiopathology)
• Cause of Death
• Child
• Child, Preschool
• Female
• Genetic Predisposition to Disease (epidemiology)
• Humans
• Incidence
• Isolated Noncompaction of the Ventricular Myocardium (genetics, mortality, physiopathology)
• Kaplan-Meier Estimate
• Male
• Multivariate Analysis
• Pediatrics
• Phenotype
• Predictive Value of Tests
• Prognosis
• Proportional Hazards Models
• Registries
• Retrospective Studies
• Survival Analysis
• United States