Steroids in Duchenne dystrophy.

The mills of God grind slowly. The medical profession are not far behind. It is almost 40 years since Drachman et al. [1] in an open study of 14 patients with Duchenne dystrophy, aged 3–10 years, for periods ranging from 1 to 28 months, concluded that steroids might have some palliative benefit, and that further studies were needed. Some 14 years later Brooke and his colleagues [2] took up the challenge and showed in a multicentric study of 33 patients, aged 5–15 years, (12 of whom had a ready lost ambulation), a definite improvement in muscle function on 1.5 mg/kg/day of prednisone, compared with the natural history of their 170 historical controls in earlier studies. At the same time DeSilva et al. [3] showed longterm benefit in some of the original Drachman cases, using age of loss of ambulation as the only criterion for assessment. They concluded, however, that the relatively short-lived beneficial effect on the muscle strength was outweighed by the not inconsiderable side effects of prolonged steroid therapy, and did not feel able to recommend steroids as a general long-term therapy for muscular dystrophy. Brooke and his colleagues [4] then went onto a randomized, double-blind, placebo-controlled trial of 103 boys aged 5–15 years, at two dosage levels of 1.5 and 0.75 mg/kg/day, over a 6 month period and found a comparable increase in muscle strength in the two prednisone groups at 1, 2 and 3 months, after which it leveled off, whereas the control group showed a steady loss of strength similar to the natural history. When Mike Brooke reviewed their cumulative data at the International Muscle Congress in Munich in 1990 [5], I was struck by the regular increase in muscle strength after initiation of therapy, followed by a plateau, and wondered whether pulsed therapy might produce similar results and avoid the not inconsiderable side effects, which they still considered at that time a constraint on recommending the treatment [6]. I tried a schedule of 10 days of therapy at 0.75 mg/kg/ day at the beginning of each month, which I thought