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Cardiomyopathies in Children and Adolescents
Quick Takes
- Among 633 individuals enrolled in a multinational registry, 47% had familial disease.
- Genetic testing identified a likely pathogenic variant in 60% of enrolled individuals.
- Outcomes differed by cardiomyopathy subtype, with patients with restrictive cardiomyopathy at highest risk for adverse cardiovascular events.
Study Questions:
What are the baseline characteristics and 1-year outcomes of children and adolescents with cardiomyopathy?
Methods:
The European Society of Cardiology EURObservational Research Programme (ESC EORP) Cardiomyopathy and Myocarditis long-term registry was used to collect prospective data on individuals aged 1 to <18 years.
Results:
A total of 633 individuals aged ≤18 years with hypertrophic (HCM; n = 388 [61.3%]), dilated (DCM; n = 206 [32.5%]), restrictive (RCM; n = 28 [4.4%]), and arrhythmogenic right ventricular cardiomyopathy (n = 11 [1.7%]) were enrolled by 23 centers in 14 countries. Median age at diagnosis was 4 years and there was a male predominance (58.8%) across all subtypes, with the exception of DCM diagnosed at <10 years of age. Familial disease was present in 47.3% of patients. Genetic testing was performed in 67.8% of patients with a pathogenic or likely pathogenic variant in 60.4%. Over a median follow-up of 12.5 months, 18 patients (3.3%) died (HCM n = 9 [2.6%], DCM n = 5 [3%], RCM n = 4 [16.0%]). Heart failure events were most frequent in RCM patients (36.0%).
Conclusions:
The authors conclude that childhood cardiomyopathies are heterogeneous in etiology and that there is a high frequency of familial disease. Outcomes differ by cardiomyopathy subtype, emphasizing the need for disease-specific evaluation and treatment.
Perspective:
This study made use of the European ESC EORP cardiomyopathy and myocarditis registry. Genetic testing in this patient population is relatively high yield, demonstrating a pathogenic variant in over 60% of patients tested. In comparison with registries in North America and Australia, HCM as opposed to DCM was the most frequent diagnosis. This may be related to the exclusion of patients <1 year of age in this registry, as DCM is more common in infants. It is also possible that HCM may be more common than previously known. Moving forward, the registry will continue to provide helpful information regarding characteristics and natural history of the various types of cardiomyopathy in children.
Clinical Topics: Heart Failure and Cardiomyopathies, Congenital Heart Disease and Pediatric Cardiology
Keywords: Cardiomyopathies, Pediatrics
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