Heart failure from heart muscle disease in childhood: a 5–10 year follow‐up study in the UK and Ireland

Abstract

AimsOur original study, the first national prospective study of new‐onset heart failure from heart muscle disease in children, showed overall 1‐year survival of 82%, and event (death or transplantation)‐free survival of 66%. This study aimed to evaluate 5 + year outcomes of this important cohort.Methods and resultsAll centres in the UK and Ireland with 1‐year event‐free survivors participated (n = 14). Anonymised data based on last hospital attendance and echocardiograms were reviewed. The investigator was blinded to outcome at the time of echo review. Of sixty‐nine 1‐year event‐free survivors, data were obtained on 64, with three lost to follow‐up and two moved abroad. There were three deaths at 2.2, 3.3 and 9.0 years after presentation and one transplant, at 5.2 years. Overall/event‐free survival was 77%/62% at 5 years and 73%/59% at 10 years, respectively. Overall and event‐free survival conditional on 1‐year survival was 94% at 5 years, and 89% at 10 years. For the 60 event‐free survivors, median (range) follow‐up duration was 9.04 (5.0–10.33) years for those still under review (n = 45), or time to discharge 5.25 (0.67–10.0) years (n = 15). Fifty‐eight were in New York Heart Association (NYHA) Class 1, and two in Class 2. Forty‐one out of sixty had normal echocardiograms at last follow‐up. Predictors of better longer‐term outcome were the same as for the original 1‐year follow‐up study, namely, younger age and higher fractional shortening measurement at presentation.ConclusionsChildren who survive the first year following their first presentation with significant heart failure from heart muscle disease have a good longer‐term outcome although there remains a small attrition rate.

Pediatric Cardiomyopathies

“…103, 104 Registries from the U.S., Australia, and Europe report transplant-free survival rates ranging from 60% to 75% within five years after diagnosis, with most events occurring within two years of presentation. 4, 10, 15, 105–107 These same registries also report that 20% to 45% of patients regain normal cardiac function during the same time period. 4, 10, 103, 105, 106 …”

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

“…In the previously mentioned prospective study of 104 children who presented with a first episode of heart failure, which was carried out in 2003, 12% required extracorporeal membrane oxygenation (ECMO) support but, as far as we can determine, none received a VAD as part of the initial presentation. 14 A follow-up report on this cohort 23 showed that 44 (42%) had normal function on echocardiogram at a median followup of 9 years and that 50% of these were on no medication. There are also data to show that children with a diagnosis of idiopathic dilated cardiomyopathy may show late recovery of function.…”

Sudden Cardiac Death in Children Affected by Cardiomyopathies: An Update on Risk Factors and Indications at Transvenous or Subcutaneous Implantable Defibrillators

“…Other recognized causes are toxic and genetic (52,53). Data show that 20% of children diagnosed with DCM normalize their systolic function within 2 years; whereas 40% die or undergo heart transplantation (54)(55)(56)(57)(58). In patients with neuromuscular, metabolic or mitochondrial disorders the underlying disease is an important determinant of outcome (1,3,59).…”