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Article
April 1991

Prednisone in Duchenne Dystrophy: A Randomized, Controlled Trial Defining the Time Course and Dose Response

Author Affiliations

From the Department of Neurology, University of Rochester (NY) (Drs Griggs and Moxley); Department of Neurology, Vanderbilt University, Nashville, Tenn (Dr Fenichel); Department of Neurology and Neurosurgery, Washington University School of Medicine (Dr Pestronk), and Division of Biostatistics, Washington University (Mr Miller). St Louis, Mo; Department of Neurology, The Ohio State University College of Medicine, Columbus (Dr Mendell); and Division of Neurology, Walter C. McKenzie Center for Health Sciences, Edmonton, Alberta (Dr Brooke).

Arch Neurol. 1991;48(4):383-388. doi:10.1001/archneur.1991.00530160047012
Abstract

• A randomized, controlled trial of daily prednisone was conducted in 99 boys (aged 5 to 15 years) with Duchenne dystrophy to define the time course of improvement and the dose response to treatment. Prednisone at 0.3 mg/kg (n=33), prednisone at 0.75 mg/kg (n = 34), and placebo (n = 32) were administered for 6 months. Patients were examined using manual muscle and myometry testing, timed functional testing, pulmonary function testing, and laboratory measurements at 10 days, 1 month, 2 months, 3 months, and 6 months of treatment. Boys treated with prednisone had stronger average muscle strength scores, than did boys treated with placebo as early as 10 days after starting therapy. At the 3-month visit, the boys in the group given 0.75 mg/kg of prednisone were significantly stronger than those in the group given 0.3 mg/kg of prednisone, indicating a dose response. At 6 months, significant side effects occurred in the group treated with 0.75 mg/kg of prednisone, including weight gain, cushingoid appearance, and excessive hair growth. Only weight gain was observed in the group taking prednisone at a dose of 0.3 mg/kg. Importantly, no side effects were evident at 10 days or 1 month of treatment, despite improvement in muscle strength and function. We conclude that prednisone produces a rapid increase in muscle strength in patients with Duchenne dystrophy and that this improvement is maximal at a prednisone dosage of 0.75 mg/kg or less.

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