Long-Term Outcomes and Prognosticators of Pediatric Primary Dilated Cardiomyopathy in an Asian Cohort

Abstract

Background: Dilated cardiomyopathy (DCM) is the most common childhood cardiomyopathy. The epidemiological profiles and prognosticators of clinical outcomes in Asian populations are not well elucidated.Methods: Data of 104 children aged <18 years with a diagnosis of primary DCM from January 1990 to December 2019 in our institutional database were retrospectively investigated. Relevant demographic, echocardiographic, and clinical variables were recorded for analysis. A P <0.05 was considered statistically significant.Results: The median age at diagnosis was 1.4 years (interquartile range = 0.3–9.1 years), and 52.9% were males. During a median follow-up duration of 4.8 years, 48 patients (46.2%) were placed on the transplantation waitlist, and 52.1% of them eventually received heart transplants. An exceptionally high overall waitlist mortality rate was noted (27.1%), which was even higher (43.5%) if the diagnostic age was <3 years. The 1-, 5-, and 10-year transplant-free were 61.1, 48.0, and 42.8%. Age at diagnosis >3 years and severe mitral regurgitation at initial diagnosis were independent risk factors for death or transplantation (hazard ratios = 2.93 and 3.31, respectively; for both, P <0.001). In total, 11 patients (10.6%) experienced ventricular function recovery after a median follow-up of 2.5 (interquartile range = 1.65–5) years. Younger age at diagnosis was associated a higher probability of ventricular function recovery.Conclusions: Despite donor shortage for heart transplantation and subsequently high waitlist mortality, our data from an Asian cohort indicated that transplant-free long-term survival was comparable with that noted in reports from Western populations. Although younger patients had exceptionally higher waitlist mortality, lower diagnostic age was associated with better long-term survival and higher likelihood of ventricular function recovery.

Pulmonary Artery Banding for Dilated Cardiomyopathy in Children: Returning to the Bench from Bedside

“…One of the main concerns about the efficacy of PAB is the possibility of having selected those patients affected by DCM in whom the LV would have recovered anyway, independently from PAB. Spontaneous healing is possible in 10–20% of patients with DCM, especially when a myocarditis-based mechanism is suspected [ 69 , 70 ]. However, functional recovery necessitates years, being extremely infrequent in the first year after the diagnosis.…”

“…However, functional recovery necessitates years, being extremely infrequent in the first year after the diagnosis. In fact, Wang et al recently described a cumulative recovery rate of 1.2%, 9.3%, and 12.1% at 1, 3, and 5 years after the diagnosis, respectively, in their pediatric cohort of patients with DCM [ 70 ]. Conversely, the significant reverse remodeling and improvement in LV function that are seen just a few months after PAB support a causative role of the procedure in promoting or accelerating myocardial healing.…”