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 We determined transplant-free survival for these children over 20 years and sought to identify the clinical characteristics at diagnosis that predicted death. Methods Children less than 18 years old with some type of DCM enrolled in the Pediatric Cardiomyopathy Registry were divided by year of diagnosis into an early cohort (1990–1999) and a late cohort (2000–2009). 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 1953 children, 1199 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 (LV) end diastolic z-scores (+4.2 vs. +4.2) and LV 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 [HR], 1.4; 95% CI, 1.04–1.9; 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.

Reference intervals and percentile curve for left ventricular outflow tract (LVOT), velocity time integral (VTI), and LVOT‐VTI‐derived hemodynamic parameters in healthy children and adolescents: Analysis of echocardiographic methods association and agreement

“…Increased heart rate 43 and impaired ventricular function are predictors of worse prognostic in childhood heart failure. 44,45 Additionally, those markers were associated with lower VTI values. 12,41 In agreement with that, in our population HR levels were negatively associated with LVOT and LVOT-VTI values ( Table 3).…”

“…Then, VTI‐derived hemodynamic parameters would be exclusively associated with “systolic load” indexes. Increased heart rate and impaired ventricular function are predictors of worse prognostic in childhood heart failure .…”

Pediatric Heart Failure: An Evolving Public Health Concern

“…Importantly, compared with the marked improvement in adult heart failure outcomes over the past 15 years, 19 there has only been a very modest improvement in survival for children with DCM since 2000. 20,21 The first hospitalization for pediatric patients with DCM is often the beginning of a period of clinical decline, progression to end stage heart failure and the need for cardiac transplantation.…”

“…In contrast, focused investigations of the molecular mechanisms underlying pediatric heart failure have started only relatively recently and provide a growing body of evidence suggesting that heart failure in children is unique from that in adults, which may, in part, account for the limited efficacy of medical therapies extrapolated from the adult heart failure experience. 17,21,28,29 Specifically, pediatric hearts that have failed secondary to DCM demonstrate unique b-adrenergic receptor adaptation, with downregulation of both b 1 and b 2 adrenergic receptors, and only b 1 adrenergic receptors are downregulated in adults with DCM. 30 In addition, the myocardium in pediatric DCM demonstrates increased cyclic adenosine monophosphate and phospholamban phosphorylation in response to chronic phosphodiesterase 3 inhibition, which is not seen in adult hearts with DCM.…”

Outcome for children following admission to hospital with a first episode of heart failure, due to heart muscle disease, in the ventricular assist device (VAD) era

“…1,2 The mortality following admission with heart failure appears to have improved over the past decades, and this is probably due to improved multidisciplinary care coupled with more widespread availability of cardiac transplantation. [3][4][5] It is known that some adults and children with suspected myocarditis may recover normal function if they can be supported through the acute phase. [6][7][8][9] It is also recognised that recovery can occur in both children 10 and adults 11 who are diagnosed with idiopathic dilated cardiomyopathy and who have no evidence of myocardial inflammation.…”