Abstract
Carvedilol and biomarkers do not have established roles in pediatric HF. We reviewed our 14-year experience to assess the role of N-terminal pro Natriuretic peptide (NTproBNP) and use of carvedilol in children with dilated cardiomyopathy (DCM).
We reviewed dosage of medications, echocardiogram measurements of ejection fraction (EF), fractional shortening corrected by age (FSz), left ventricular end diastolic diameter corrected by BSA (LVEDDz) and NTproBNP to assess outcome and response to treatment.
There were 106 children with DCM (58 male). Mean age at presentation was 7.4±6.5 years. Mean EF and FSz at presentation were 28.9±10 and −8.1±6.7. Carvedilol and ACE inhibitors were used in combination in 72% of patients. Digoxin was used in symptomatic patients. Average dose (mg/kg/day) of carvedilol was 0.95 (range 0.05-1.7), enalapril 0.64 (0.08-1.2) and captopril 3.4 (0.45-6.4). Average follow up was 6.2±5.5 years. Death/transplant (DT) occurred in 45% of children; 40% of those occurred within one year of presentation. In the survivors, EF and FSz improved from 29.3±9.6 to 53.8±9 (p<0.001) and −8.3±7.4 to −3.1±3.5 (p=0.002); in 80% of the survivors EF improved to ≥50%. LVEDDz decreased from 4.5±2.9 to 1.7±2.1 (p<0.001). NTproBNP was available in 46 patients; 82% of the patients with DT had NTproBNP≥1000 pg/ml. In all but 2 of the living or non-transplanted survivors, NTproBNP decreased below 1000 pg/ml. Only 3 patients did not tolerate carvedilol, 2 because of asthma and 1 for worsening heart failure. Three patients experienced one episode of hypoglycemia requiring hospitalization, but there was no need to discontinue medication.
Mortality and transplant remain common outcomes for children with DCM, particularly in the first year following presentation. Carvedilol and ACE inhibitors are safely tolerated with long-term use, and at higher doses than previously reported. Hypoglycemia may be a serious side effect that can be avoided by taking carvedilol with food. Persistent NTproBNP≥1000 may indicate need for transplantation.
