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

Neurology. 1993 Mar;43(3 Pt 1):520-7. doi: 10.1212/wnl.43.3_part_1.520.


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.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Azathioprine / administration & dosage*
  • Azathioprine / adverse effects
  • Body Height
  • Child
  • Child, Preschool
  • Double-Blind Method
  • Drug Therapy, Combination
  • Humans
  • Male
  • Muscular Dystrophies / drug therapy*
  • Muscular Dystrophies / physiopathology
  • Placebos
  • Prednisone / administration & dosage*
  • Prednisone / adverse effects
  • Time Factors
  • Weight Gain


  • Placebos
  • Azathioprine
  • Prednisone