Abstract • We previously reported the results of a randomized, double-blind 6-month trial of prednisone therapy in which 102 boys aged 5 to 15 years with Duchenne muscular dystrophy received daily doses of 1.5 and 0.75 mg/kg per day and were compared with those receiving placebo. The strength and function in both prednisone-treated groups improved equally and were significantly better than in the placebo group. To compare alternate-day and daily dosing of prednisone with respect to benefits and adverse side effects, the placebo group was started on alternate-day prednisone therapy, and the treatment group regimens were changed to equivalent doses of alternate-day prednisone without breaking the double-blind nature. At the end of 6 months, the group that was changed from daily to alternate-day therapy had declined in strength back to levels observed 12 months previously, at the start of daily therapy. The group in which alternate-day therapy was started showed a significant improvement in strength at 3 months, similar in magnitude to the response of boys treated with daily therapy. However, their strength declined significantly in the subsequent 3 months compared with boys who received daily therapy. The frequency of side effects was not significantly different for alternate-day therapy compared with daily therapy. We conclude that alternate-day prednisone therapy effectively increases strength but does not sustain the improvement to the same extent as daily therapy or mitigate side effects. References 1. Koenig M, Hoffman EP, Bertelson CJ, et al. Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals . Cell . 1987;50:509-517.Crossref 2. Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus . Cell . 1987;51:919-928.Crossref 3. Rowland LP. Clinical concepts of Duchenne muscular dystrophy: the impact of molecular genetics . Brain . 1988;111:479-495.Crossref 4. Mendell JR, Moxley RT, Griggs RC, et al. Randomized double-blind controlled trial of prednisone in Duchenne muscular dystrophy . N Engl J Med . 1989;320:1592-1597.Crossref 5. Brooke MH, Griggs RC, Mendell JR, et al. Clinical trial in Duchenne dystrophy, I: the design of the protocol . Muscle Nerve . 1981;4:186-197.Crossref 6. Brooke MH, Fenichel GM, Griggs RC, et al. Clinical investigation in Duchenne dystrophy, II:determination of the 'power' of therapeutic trials based on the natural history . Muscle Nerve . 1983;6:91-103.Crossref 7. Marascuilo LA, McSweeney M. Nonparametric and Distribution-Free Methods for the Social Sciences . Monterey, Calif: Brooks/Cole Publishing Co; 1977:167-169. 8. SAS Institute Inc. SAS/STAT User's Guide, Release 6.03 Edition . Cory, NC: SAS Institute Inc; 1988. 9. Mendell JR, Province MA, Moxley RT, et al. Clinical investigation of Duchenne muscular dystrophy: a methodology for therapeutic trials based on natural history controls . Arch Neurol . 1987;44:808-811.Crossref 10. Harter JG, Reddy WJ, Thorn GW. Studies on an intermittent corticosteroid dosage regimen . N Engl J Med . 1963;269:591-596.Crossref 11. Fauci AS. Alternate-day corticosteroid therapy . Am J Med . 1978;64:729-731.Crossref 12. Fauci AS, Dale DC, Balou JE. Glucocorticosteroid therapy: mechanism of action and clinical considerations . Ann Intern Med . 1986;84:304-315.Crossref 13. Forbes GB, Bruining GJ. Urinary creatinine excretion and lean body mass . Am J Clin Nutr . 1976;29:1359-1366. 14. Griggs RC, Forbes GB, Moxley RT, et al. The assessment of muscle mass in progressive neuromuscular disease . Neurology . 1983;33:158-165.Crossref 15. Fenichel GM, Pestronk FA, Miller JP, et al. Prednisone slows strength decline in Duchenne muscular dystrophy: two-year observation . Neurology . 1991;41( (suppl 1) ):166. Abstract 182S. 16. Burrow KL, Coovert D, Klein CJ, et al. Dystrophin analysis following prednisone treatment of Duchenne muscular dystrophy . Neurology . 1990;40 ( (suppl 1) ):206. Abstract 321P. 17. Kissel JT, Burrow K, Rammohan KW, et al. Mononuclear cell analysis of muscle biopsies in prednisone-treated Duchenne muscular dystrophy . Neurology . 1990;40( (suppl 1) ):412. Abstract 1076S.
Archives of Neurology – American Medical Association
Published: Jun 1, 1991