title: Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months)
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.
authors.affiliation: Department of Neurology, University of Rochester, NY.
authors.forename: R C
authors.initials: RC
authors.lastname: Griggs
authors.forename: R T
authors.initials: RT
authors.lastname: Moxley
authors.forename: J R
authors.initials: JR
authors.lastname: Mendell
authors.forename: G M
authors.initials: GM
authors.lastname: Fenichel
authors.forename: M H
authors.initials: MH
authors.lastname: Brooke
authors.forename: A
authors.initials: A
authors.lastname: Pestronk
authors.forename: J P
authors.initials: JP
authors.lastname: Miller
authors.forename: V A
authors.initials: VA
authors.lastname: Cwik
authors.forename: S
authors.initials: S
authors.lastname: Pandya
authors.forename: J
authors.initials: J
authors.lastname: Robison
mesh_terms: D000293:Adolescent; D001379:Azathioprine / Q000008:administration & dosage* / Q000009:adverse effects; D001827:Body Height; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D004359:Drug Therapy, Combination; D006801:Humans; D008297:Male; D009136:Muscular Dystrophies / Q000188:drug therapy* / Q000503:physiopathology; D010919:Placebos; D011241:Prednisone / Q000008:administration & dosage* / Q000009:adverse effects; D013997:Time Factors; D015430:Weight Gain
chemical_list: D010919:Placebos; D001379:Azathioprine; D011241:Prednisone