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A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy

From the Departments of Neurology (Drs. Mendell and Kissel and W.M. King), Ohio State University College of Medicine; the University of Rochester and Strong Memorial Hospital (Drs. Griggs and Moxley and S. Pandya), NY; Vanderbilt University School of Medicine (Dr. Fenichel and V. Robison); Washington University School of Medicine (Dr. Pestronk and J. Florence), St. Louis, MO; and Bio-Technology General Corp. (Drs. Kramer and Sheng and H. Wang).

Address correspondence and reprint requests to Dr. Gerald Fenichel, Neurology, 2100 Pierce Avenue, Nashville, TN 37212; e-mail: gerald.fenichel{at}mcmail.vanderbilt.edu

BACKGROUND: A pilot study suggested that oxandrolone, an anabolic steroid, improved strength in boys with Duchenne dystrophy (DD) and indicated the need for a more definitive study.

METHODS: A 6-month, randomized, double-blind, placebo-controlled study of oxandrolone in boys with an established diagnosis of DD, using the change from baseline to 6 months in the average muscle strength score (MMT) as the primary efficacy measure.

RESULTS: The mean change from baseline for the oxandrolone group was +0.035 and that for the placebo group was -0.140. Although the oxandrolone group did not get worse and the placebo patients showed some deterioration in strength, the difference was not significant (p = 0.13). The average of the four quantitative muscle tests (QMT) showed a significant improvement in the oxandrolone-treated boys as compared with placebo. No adverse reactions attributable to oxandrolone were recorded.

CONCLUSIONS: Although oxandrolone did not produce a significant change in the average manual muscle strength score as compared with placebo, the mean change in QMT was significant. Because oxandrolone is safe, accelerates linear growth, and may have some beneficial effect in slowing the progress of weakness, it may be useful before initiating corticosteroid therapy.

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