New-Onset Heart Failure Due to Heart Muscle Disease in Childhood

Abstract

Background— We undertook the first prospective, national, multicenter study to describe the incidence and outcome of heart muscle disease–induced heart failure in children. Methods and Results— Data were collected on patients admitted to a hospital through 2003 with a first episode of heart failure in the absence of congenital heart disease. All 17 pediatric cardiac centers in the United Kingdom and Ireland participated. Follow-up data were obtained to a minimum of 1 year. The incidence was 0.87/100 000 population <16 years (n=104; 53 girls; 95% confidence interval 0.71 to 1.05 per 100 000). Median age at presentation was 1 year, with 82% in New York Heart Association class III to IV. Causes of heart failure included dilated cardiomyopathy (50 idiopathic, 8 familial), probable myocarditis (23), occult arrhythmia (7), anthracycline toxicity (5), metabolic disease (4), left ventricular noncompaction (3), and other (4). Overall 1-year survival was 82%, and event (death or transplantation)-free survival was 66%. Regression analysis showed older age and reduced systolic function on admission echocardiogram increased the event risk. Only 8% of event-free survivors (n=69) remained in New York Heart Association class III to IV, but 35 required readmission during the study period, and all but 8 remained on medication. Conclusions— This first national prospective study of new-onset heart failure in children has shown an incidence of 0.87/100 000. Multivariable analysis of survival data indicates a better outcome for younger children and for those with better systolic function at presentation, but overall, one third of children die or require transplantation within 1 year of presentation.

Loss ofProx1in striated muscle causes slow to fast skeletal muscle fiber conversion and dilated cardiomyopathy

“…puberty (6-8 wk), in line with a second peak of human DCM that occurs during early adolescence (38). There is also a subset of patients that develop DCM in later life following earlier presentation with congenital heart defects, such as incomplete septation and failed myocardial compaction (23,39).…”

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

“…The same methodology was used as in the original study, to ensure a valid comparison 4. As in the original study, when all diagnostic groups were included in the multivariable model, only older age at presentation predicted death or transplantation significantly.…”

“…As previously published,4 this comprised 104 children <16 years of age who required hospital admission from January–December 2003 for new‐onset heart failure from heart muscle disease. The cohort included primary cardiomyopathies, those secondary to neuromuscular or metabolic disease, anthracycline toxicity and previously undiagnosed arrhythmia.…”

“…Two studies from the USA have used retrospective database analyses to look at heart failure‐related intensive care admissions in children with cardiomyopathy2 and heart failure‐related hospitalizations,3 but this latter study included children with congenital heart disease. Our original study involved prospectively collecting data on all children <16 years of age who were admitted to hospital in the UK and Ireland with a new diagnosis of heart failure from heart muscle disease through one calendar year, from January–December 2003 4. The impetus for this study arose from a question posed by the UK Department of Health National Speciality Commissioning Group, who commission heart transplantation and mechanical support in the National Health Service, as to the incidence of symptomatic heart failure from heart muscle disease in children.…”

Heart Failure in Children

“…2,3 Unfortunately, heart failure class at presentation is a poor predictor of worsening clinical outcome. 4,5 The scenario of a child with few symptoms who suddenly develops decompensated heart failure is well known and highlights the limitations of heart failure class as a predictor or outcome in children.…”