Survival Without Cardiac Transplantation Among Children With Dilated Cardiomyopathy

Abstract

Background: Studies of children with dilated cardiomyopathy (DCM) have suggested that improved survival has been primarily due to utilization of heart transplantation.

Objectives: This study sought to determine transplant-free survival for these children over 20 years and identify the clinical characteristics at diagnosis that predicted death.

Methods: Children <18 years of age with some type of DCM enrolled in the Pediatric Cardiomyopathy Registry were divided by year of diagnosis into an early cohort (1990 to 1999) and a late cohort (2000 to 2009). Competing risks and multivariable modeling were used to estimate the cumulative incidence of death, transplant, and echocardiographic normalization by cohort and to identify the factors associated with death.

Results: Of 1,953 children, 1,199 were in the early cohort and 754 were in the late cohort. Most children in both cohorts had idiopathic DCM (64% vs. 63%, respectively). Median age (1.6 vs. 1.7 years), left ventricular end-diastolic z-scores (+4.2 vs. +4.2), and left ventricular fractional shortening (16% vs. 17%) at diagnosis were similar between cohorts. Although the rates of echocardiographic normalization (30% and 27%) and heart transplantation (24% and 24%) were similar, the death rate was higher in the early cohort than in the late cohort (18% vs. 9%; p = 0.04). Being in the early cohort (hazard ratio: 1.4; 95% confidence interval: 1.04 to 1.9; p = 0.03) independently predicted death.

Conclusions: Children with DCM have improved survival in the more recent era. This appears to be associated with factors other than heart transplantation, which was equally prevalent in both eras. (Pediatric Cardiomyopathy Registry [PCMR]; NCT00005391).

Keywords: cardiomyopathy; echocardiography; heart failure; pediatrics; transplantation.

Conflict of interest statement

Disclosures: The authors report no apparent competing interests.

Copyright © 2017 American College of Cardiology Foundation. Published by Elsevier Inc. All rights reserved.

Figures

Figure 1. Estimated A) time to death (log rank p ≤ 0.001) and B) time to heart transplantation (log rank p = 0.12) in 1953 children with dilated cardiomyopathy, by cohort

Follow-up times were censored at time of heart transplantation, where applicable.

Figure 1. Estimated A) time to death (log rank p ≤ 0.001) and B) time to heart transplantation (log rank p = 0.12) in 1953 children with dilated cardiomyopathy, by cohort

Follow-up times were censored at time of heart transplantation, where applicable.

Figure 2. Estimated cumulative incidence rates for echocardiographic normalization in the presence of the competing risk for death or transplant among 1953 children with dilated cardiomyopathy. A) Early cohort (diagnosed between 1990 and 1999). B) Late cohort (diagnosed between 2000 and 2009)

At any given time, the probabilities associated with the 4 states sum to 1.0. Although the rates of echocardiographic normalization and transplantation were similar between cohorts (p = 0.22 and p = 0.35, respectively), the early cohort had a higher death rate (p = 0.04).

Figure 2. Estimated cumulative incidence rates for echocardiographic normalization in the presence of the competing risk for death or transplant among 1953 children with dilated cardiomyopathy. A) Early cohort (diagnosed between 1990 and 1999). B) Late cohort (diagnosed between 2000 and 2009)

At any given time, the probabilities associated with the 4 states sum to 1.0. Although the rates of echocardiographic normalization and transplantation were similar between cohorts (p = 0.22 and p = 0.35, respectively), the early cohort had a higher death rate (p = 0.04).

Figure 3. Estimated cumulative incidence rates of echocardiographic normalization in the presence of the competing risk for death or transplant for children with dilated cardiomyopathy, by type. A) Early cohort (diagnosed between 1990 and 1999); B) Late cohort (diagnosed between 2000 and 2009)

At any given time, the probabilities associated with the 4 states sum to 1.0. Rates of echocardiographic normalization, death and transplantation did not differ by cohort among children with familial DCM, idiopathic DCM, or myocarditis. Although the rates of death and transplantation were similar between cohorts among children with neuromuscular disease, the late cohort had a higher rate of echocardiographic normalization (p

Figure 3. Estimated cumulative incidence rates of echocardiographic normalization in the presence of the competing risk for death or transplant for children with dilated cardiomyopathy, by type. A) Early cohort (diagnosed between 1990 and 1999); B) Late cohort (diagnosed between 2000 and 2009)

At any given time, the probabilities associated with the 4 states sum to 1.0. Rates of echocardiographic normalization, death and transplantation did not differ by cohort among children with familial DCM, idiopathic DCM, or myocarditis. Although the rates of death and transplantation were similar between cohorts among children with neuromuscular disease, the late cohort had a higher rate of echocardiographic normalization (p

Central Illustration. Survival in Pediatric Dilated Cardiomyopathy: Estimated time to death and time to heart transplantation among 1,953 children with dilated cardiomyopathy, by type

Follow-up times were censored at the time of heart transplantation, where applicable. FDCM: familial dilated cardiomyopathy; IDCM: idiopathic dilated cardiomyopathy, NM: neuromuscular disease