Growth hormone treatment in boys with Duchenne muscular dystrophy and glucocorticoid-induced growth failure.

This study evaluated efficacy and safety of growth hormone treatment in Duchenne muscular dystrophy boys with glucocorticoid-induced growth failure. We reviewed 39 consecutive boys (average age 11.5years; 32 ambulatory) treated with growth hormone for 1year during a four-year period. Boys were on long-term daily deflazacort or prednisone (mean duration 5±2.2years; dosing regimen prednisone 0.75mg/kg/day equivalent). Primary outcomes were growth velocity and height-for-age z-scores (height SD) at 1year. Height velocity increased from 1.3±0.2 to 5.2±0.4cm/year on growth hormone (p<0.0001). Pre-growth hormone decline in height SD (−0.5±0.2SD/year) stabilized at height SD −2.9±0.2 on growth hormone (p<0.0001). The rate of weight gain was unchanged, at 2.8±0.6kg/year pre-growth hormone and 2.6±0.7kg/year at 1year. Motor function decline was similar pre-growth hormone and at 1year. Cardiopulmonary function was unchanged. Three experienced side effects. In this first comprehensive report of growth hormone in Duchenne muscular dystrophy, growth hormone improved growth at 1year, without detrimental effects observed on neuromuscular and cardiopulmonary function.