Deflazacort treatment of Duchenne muscular dystrophy

Objective: We report the long-term effects on muscle strength and side effects with deflazacort in Duchenne muscular dystrophy (DMD). Study design: Boys with DMD between the ages of 7 and 15 years were reviewed retrospectively; 30 had been treated with deflazacort, and 24 had not. muscle function, pulmonary function, and side effects were compared. Results: The boys not treated with deflazacort stopped walking at 9.8 +/- 1.8 years. Seven of 30 treated boys had stopped walking at 12.3 +/- 2.7 years (P < .05), and of the 23 boys who were still walking, 21 were older than 10 years. Pulmonary function (percent predicted functional vital capacity) was significantly greater in treated boys at 15 years (88% +/- 18%) than in boys not treated (39% +/- 20%) (P < .001). Between 9 and 15 years, treated boys were shorter. Between 9 and 13 years, treated boys weighed less. After 13 years the treated boys maintained their weight, whereas boys not treated lost weight. Asymptomatic cataracts developed in 10 of 30 boys who received deflazacort. Other potential side effects of deflazacort such as hypertension, glucosuria, acne, infection, or bruising were not more common. Conclusions: We conclude that deflazacort can preserve gross motor and pulmonary function in boys with DMD with limited side effects.