P.11.15 Recombinant human insulin-like growth factor-I (IGF-I) therapy in Duchenne Muscular Dystrophy (DMD): A 6-month prospective randomized controlled trial

Abstract

Glucocorticoids (GC) delay motor decline in DMD, but cause significant endocrine adverse effects. IGF-I offers potential as a therapeutic agent; it may improve or preserve muscle function and counter GC effects of growth failure and insulin resistance. To determine if IGF-I therapy (1) improves or preserves muscle function and (2) improves linear growth in DMD. Prospective randomized controlled trial of IGF-I therapy in pre-pubertal, ambulatory, GC-treated DMD boys (n = 17) compared to controls (GC alone, n = 21). IGF-I 160 mcg/kg was given daily subcutaneously for 6 months. Primary motor outcome was 6-min walk distance (6MWD), and endocrine outcomes were height velocity and change in height SD score (ΔHtSDS). Other outcomes included changes in timed motor tests, cardiopulmonary function, insulin sensitivity and safety. The difference of 6MWD between control and IGF-I groups at 6 months was 8.5 ± 7.0 m (mean ± SEM, p > 0.5). There were no significant differences between groups for changes in cardiopulmonary and other motor functional outcomes. Height velocity in IGF-I treated subjects was double that of controls at 6 months (6.5 ± 0.4 vs. 3.3 ± 0.3 cm/yr, p In this first study of IGF-I therapy in DMD boys, 6 months of daily IGF-I significantly increased height velocity and HtSDS compared to controls, but there was no difference in motor functional outcomes.