What is the clinical evidence from this study?

Study design: Cohort. Population: Childhood cardiomyopathy (DCM, HCM, LVNC, RCM) (n=317). Intervention: NYHA/Ross class III or IV vs. NYHA/Ross class I or II. Primary outcome: Mortality in dilated cardiomyopathy (HR 3.77, 95% CI 1.3-10.9, p=0.014).

October 6, 2023Open Access

The clinical profile, genetic basis and survival of childhood cardiomyopathy: a single- center retrospective study

Resultado clave

NYHA/Ross class III or IV was a significant predictor of mortality in children with dilated cardiomyopathy (HR 3.77) and restrictive cardiomyopathy (HR 8.65), which had the highest overall mortality.

Diseño del estudio

Tipo

Cohort (n=317)

Multicéntrico

PICO estructurado

Población

317 children aged 18 years or younger with cardiomyopathy (DCM, HCM, LVNC, or RCM) were retrospectively evaluated for clinical characteristics and survival outcomes over a 10-year period.

Resultado

Mortality / survivalhard clinical

Childhood cardiomyopathy, particularly restrictive cardiomyopathy, is associated with high mortality, and NYHA/Ross class III or IV is a strong predictor of adverse outcomes.

Resultado numérico

Hazard Ratio: 3.77 (95% CI 1.3–10.9)

valor p: p=0.014

Limitaciones

Relatively small sample size recruited from a single medical center
Diagnosis based on clinical and echocardiographic findings without cardiac catheterization or histological examination
Genetic analysis was only performed in a subset of patients
Retrospective study design with potential inherent biases

Resumen

Abstract Background: Cardiomyopathy (CM) is a heterogeneous group of myocardial diseases in children. This study aimed to identify demographic features, clinical presentation and prognosis of children with CM. Methods: Clinical characteristics and prognostic factors associated with mortality were evaluated by Cox proportional hazards regression analyses. Genetic testing was also conducted on a portion of patients. Results: Among the 317 patients, 40.1%, 25.2%, 24.6%, and 10.1% were diagnosed with dilated cardiomyopathy (DCM), hypertrophic cardiomyopathy (HCM), left ventricular noncompaction cardiomyopathy (LVNC) and restrictive cardiomyopathy (RCM), respectively. The most common symptom observed was dyspnea (84.2%). Except for HCM, the majority of patients were classified as NYHA/Ross class III or IV. The five- and ten- year survival rates were 75.5% and 60.1%, 67.3% and 56.1%, 74.1% and 57.2%, and 51.1% and 41.3% in DCM, HCM, LVNC, and RCM, respectively. Survival was inversely related to NYHA/Ross class III or IV in patients with DCM, HCM and RCM. Out of 42 patients, 32 were reported to carry gene mutations. Conclusions: This study demonstrates that CM, especially RCM, is related to a high incidence of death. NYHA/Ross class III or IV is a predictor of mortality in the patients and gene mutations may be a common cause.

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