Recombinant Human Growth Hormone Treatment for Dilated Cardiomyopathy in Children

Abstract

In this prospective, single-center, randomized, partially blinded, crossover trial, recombinant human GH was administered to 8 pediatric patients with stable chronic heart failure secondary to DCM. Because of unanticipated difficulty enrolling eligible patients, the study was underpowered to detect changes in our primary outcome measure of the magnitude we projected. Nevertheless, we did observe several notable cardiovascular effects of GH treatment, including a trend toward improved LV ejection fraction during the course of GH treatment and significantly improved LV SF, SF z score, and LV end systolic stress z score 6 months after discontinuation of GH treatment (relative to baseline values). Given the fact that levels of IGF-1, the primary myocardial effector of GH signaling, remained significantly higher 6 months after GH treatment than at baseline, the improvement in LV functional indices 6 months after discontinuation of therapy may represent progression or perpetuation of a GH treatment effect. In addition to its cardiovascular effects, GH therapy was associated with significant acceleration of somatic growth. The benefits of GH were not associated with significant attributable side effects, although 2 patients developed progressive LV dysfunction during the study and underwent cardiac transplantation.