Growth hormone therapy for children with Duchenne muscular dystrophy and glucocorticoid induced short stature

Abstract

Objective: To evaluate the outcome of recombinant human Growth Hormone (rhGH) therapy in patients with Duchene Muscular Dystrophy (DMD) and glucocorticoid treatment with compromised growth.

Design: Four DMD patients on Deflzacort 0.6-0.85 mg/kg/day or prednisolone 0.625 mg/kg/day recieved rhGH (0.24 mg/kg/week) for 6-18 months. Primary outcomes were Growth velocity and Height for age Z-scores (Height SD).

Results: Growth velocity increased from 0 to 3.25 cm/year prior to GH therapy to 3.3-7.8 cm/year over a period of 6-18 months. The typical Height SD decline in DMD was reversed in two patients and blunted in one. No adverse events or deterioration in cardiac or respiratory parameters were associated with the rhGH treatment.

Conclusions: rhGH appears to be safe and efficient in promoting growth of patients with glucocorticoid induced growth failure in DMD.

Keywords: Duchenne muscular dystrophy; Glucocorticoids; Growth failure; Growth hormone; Short stature.