A novel treatment regimen for Duchenne muscular dystrophy

Abstract

Duchenne muscular dystrophy (DMD) is the most common, X-linked genetic, skeletal muscle disease, with various regimens of treatment. The objective of this study was to determine the safety and efficacy of a novel treatment regimen for this disease. Thirty boys with DMD were administered prednisone according to the following regimen: in the first year, 1.5 mg/kg/day for the first 3 months, 1.0 mg/kg/day for the next 3 months, 0.75 mg/kg/day for the next 3 months, and 0.5 mg/kg/day for the last 3 months. In the second year, prednisone was administered 0.5 mg/kg on the alternate day for 12 months. The muscle strength (Medical Research Council sum score and Gower's sign), serum enzymes (creatine kinase, creatine kinase isoenzyme-2, and lactate dehydrogenase), pulmonary function (forced vital capacity, maximum voluntary ventilation), body weight, height, and BMI were determined before treatment and 3, 6, 9, 12, and 24 months after treatment. The results showed that the patients' mean Medical Research Council sum score increased from 46.1 at the baseline to 53.6 at 12 months and was maintained at 24 months. Gower's sign disappeared in 22 (73.3%) patients at 12 months and 21 (70.0%) at 24 months. The serum levels of creatine kinase, creatine kinase isoenzyme-2, and lactate dehydrogenase decreased and pulmonary function improved after 24 months of treatment. Significantly increased weight gain, osteoporosis, and cushingoid features were not observed. Our results suggested that this novel prednisone regimen for DMD has similar efficacy and safety as other regimens.