The Pediatric Carvedilol Study trial offers a unique opportunity to investigate patient characteristics and risk factors associated with death or transplant in a population of children with moderate heart failure symptoms. The database from the Pediatric Carvedilol Study trial includes 157 children enrolled with symptomatic heart failure (NYHA II–IV) and ejection fraction (EF)<40%. For this analysis, the patients were divided into by outcome into 2 groups: those who died or underwent heart transplant during the study period (D/HTx) and those who did not (non-D/HTx). Baseline variables were compared using standard parametric and non-parametric testing. Multivariate analysis was performed using logistic regression
Results: Of the 157 children, the median age at enrollment was 3 yrs (0.2–17.8yrs). Fifty-two percent were male, and 116/157 (74%) had a left ventricle (LV) as the systemic ventricle. All pts except 3 were NYHA/Ross class II-III, with a median EF of 25.7%, median BNP of 111, and median LVEDD z-score of 6.04. Overall incidence of D/HTx in the enrolled patients over the study period was 30/157 (19%), with 12 deaths and 18 transplants. Risk factors for D/HTx were older age (8.1yrs vs 2.1 yrs, p< 0.006), higher BNP levels at enrollment (250 vs 80, p<0.003) and worse AVV regurgitation (p<0.012), but not diagnosis of congenital heart disease, ventricular morphology, or qualitative assessment of systemic ventricular dysfunction. In the subgroup of patients with a systemic LV, the D/HTx group had a significantly larger EDD z-score (7.7 vs 5.8, p=0.05), but no difference in ejection fraction (25.9 vs 25.6, p=0.8) compared to the non D/HTx. In a multi-variate model after adjusting for age, LV EDD z-score and BNP at enrollment were significantly related to risk for D/HTx. For this pediatric population with chronic symptomatic heart failure, risk factors for death or transplant included older age, higher BNP, and AVVR. In the subgroup of patients with a systemic left ventricle, larger LV EDD z-score, but not lower EF, was significantly related to short term risk for death or transplant.
Risk factors for asymptomatic echocardiographic abnormalities that predict symptomatic heart failure
