Duchenne dystrophy: Randomized, controlled trial of prednisone (18 months) and azathioprine (12 months)

Robert C. Griggs, R. T. Moxley, J. R. Mendell, G. M. Fenichel, M. H. Brooke, A. Pestronk, J. P. Miller, V. A. Cwik, S. Pandya, J. Robison, W. King, L. Signore, J. Schierbecker, J. Florence, N. Matheson-Burden, B. Wilson

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164 Scopus citations

Abstract

Prednisone has been shown to improve strength in Duchenne dystrophy. Azathioprine often benefits corticosteroid-responsive diseases and can reduce the dose of prednisone needed. The present study reports a randomized, controlled trial of prednisone and azathioprine designed to assess the longer-term effects of prednisone and to determine whether azathioprine alone, or in combination with prednisone, improves strength. Ninety-nine boys (aged five to 15 years) with Duchenne dystrophy were randomized to one of three groups: (I) placebo; (II) prednisone 0.3 mg/kg/d; or (III) prednisone 0.75 mg/kg/d. After 6 months, azathioprine 2 to 2.5 mg/kg/d was added in groups I and II and placebo added in group III. The study showed that the beneficial effect of prednisone (0.75 mg/kg/d) is maintained for at least 18 months and is associated with a 36% increase in muscle mass. There was weight gain, growth retardation, and other side effects. Azathioprine did not have a beneficial effect. This study suggests that prednisone's beneficial effect is not due to immunosuppression.

Original languageEnglish
Pages (from-to)520-527
Number of pages8
JournalNeurology
Volume43
Issue number3
DOIs
StatePublished - Mar 1993

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