Survival Without Cardiac Transplantation Among Children With Dilated Cardiomyopathy
Key result
Children diagnosed with dilated cardiomyopathy in the early cohort (1990-1999) were 40% more likely to die than those in the late cohort (2000-2009) (HR 1.4), despite similar rates of heart transplantation.
Why the study?
Studies of children with DCM have suggested that improved survival has been primarily due to heart transplantation, prompting evaluation of transplant-free survival over 20 years and predictors of death.
Does diagnosis in a more recent era (2000-2009) improve transplant-free survival in children with dilated cardiomyopathy compared to an earlier era (1990-1999)?
Key Points
- To determine 20-year trends in transplant-free survival and identify baseline clinical predictors of mortality among children with dilated cardiomyopathy.
- Analyzed 1953 children aged less than 18 years with dilated cardiomyopathy enrolled in the Pediatric Cardiomyopathy Registry, stratified into an early cohort (1990–1999, n=1199) and a late cohort (2000–2009, n=754).
- Calculated cumulative incidence of death, heart transplantation, and echocardiographic normalization using competing risks and multivariable modeling across cohorts.
- All-cause mortality was significantly higher in the early cohort compared to the late cohort (18% vs. 9%; p = 0.04), despite similar rates of heart transplantation (24% vs. 24%) and echocardiographic normalization (30% vs. 27%).
- Diagnosis in the early cohort independently predicted death on multivariable analysis (hazard ratio [HR], 1.4; 95% CI, 1.04–1.9; p = 0.03).
- Baseline median age (1.6 vs. 1.7 years), left ventricular fractional shortening (16% vs. 17%), and left ventricular end-diastolic z-scores (+4.2 vs. +4.2) were similar between early and late cohorts at diagnosis.
Study Design
Type
Cohort (n=1,953)
Multicenter
Yes
Structured PICO
Does diagnosis in a more recent era (2000-2009) improve transplant-free survival in children with dilated cardiomyopathy compared to an earlier era (1990-1999)?
P
Population
1,953 children under 18 years old with pure dilated cardiomyopathy enrolled in the Pediatric Cardiomyopathy Registry, followed for a median of 1.0 to 1.6 years to compare outcomes between 1990-1999 and 2000-2009 cohorts.
E
Exposure
Diagnosis and management in the late cohort era (2000-2009)
C
Comparator
Diagnosis and management in the early cohort era (1990-1999)
O
Outcome
Cumulative incidence of death, heart transplant, persistently abnormal echocardiogram, and echocardiographic normalizationhard clinical
Main Result
Hazard Ratio: 1.4 (95% CI 1.04–1.89)
Absolute Event Rate: 18% vs 9%
p-value: p=0.028
Limitations
- Observational design does not allow associations between survival and various therapeutic modalities to be assessed.
- Limited information on clinical care after diagnosis, such as the use and duration of inotropic support, mechanical ventilation, and mechanical circulatory support.
- Data on potentially important echocardiographic variables, such as mitral or tricuspid regurgitation, were not available for most children.
- Data on the use of beta-blockers and angiotensin-converting enzyme inhibitors were not collected in more than half the children.
- Limited information on clinical care after diagnosis, such as use of inotropic support, mechanical ventilation, and mechanical circulatory support.
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Key expert perspectives
Captured external expert commentary on this paper, strongest first. Original sources are linked where available.
“We have been able to reduce failure of medical management by about 50% which translates to a large number of children with dilated cardiomyopathy who are alive without heart transplant compared to children with dilated cardiomyopathy at the same centers in the past who would have died. When you consider that in this population half the children who would have died are living this is truly incredible.”
“Children with dilated cardiomyopathy have better survival in the more recent era, which appears to be associated with factors other than availability of transplantation as that was equally prevalent in both eras.”
“Further research including documentation of medical therapy and use of mechanical circulatory support will be necessary to understand the changes in survival over time seen in this study.”
Implication
Survival without heart transplantation has significantly improved for children with dilated cardiomyopathy in the 2000s compared to the 1990s, suggesting that advances in medical and device therapies are driving better outcomes.
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.
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Cite This Study
Singh et al. (2017) conducted a cohort in Dilated cardiomyopathy (n=1,953). Diagnosis in the early cohort (1990-1999) vs. Diagnosis in the late cohort (2000-2009) was evaluated on Death without heart transplant (HR 1.4, 95% CI 1.04-1.89, p=0.028). Children diagnosed with dilated cardiomyopathy in the early cohort (1990-1999) were 40% more likely to die than those in the late cohort (2000-2009) (HR 1.4), despite similar rates of heart transplantation.