AIMS: Our 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 RESULTS: All 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 9.0years after presentation and one transplant, at 5.2years. Overall/event-free survival was 77%/62% at 5years and 73%/59% at 10years, respectively. Overall and event-free survival conditional on 1-year survival was 94% at 5years, and 89% at 10years. 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.
CONCLUSIONS: Children 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.