A Cochrane review [Abstract] 1 included 12 studies with a total of 667 subjects with Duchenne muscular dystrophy. Only one RCT (n=28) used prolongation of time to loss of walking as an outcome measure, with deflazacort 2 mg/kg on alternate days as the active intervention, but data were not adequate for drawing conclusions. Meta-analyses showed that corticosteroids (0.75 mg/kg/day prednisone or prednisolone) improved muscle strength and function 0.52 units more on Medical Research Council (MRC) strength score vs. placebo over 6 months (0.33 more to 0.71 more, 3 trials, n=147). Evidence from single trials showed 0.75 mg/kg/day superior to 0.3 mg/kg/day on most strength and function measures, with little evidence of further benefit at 1.5 mg/kg/day. Improvements were seen in time taken to rise from the floor (Gowers' time), timed walk, 4-stair climbing time, ability to lift weights, leg function grade, and forced vital capacity. One RCT (n = 66) reported better strength, function and quality of life with daily 0.75 mg/kg/day prednisone at 12 months. One RCT (n = 28) showed that deflazacort stabilised muscle strength vs. placebo at 2 years, but timed function test results were too imprecise for conclusions.One double-blind RCT (n = 64) compared daily prednisone (0.75 mg/kg/day) with weekend-only prednisone (5 mg/kg/weekend day), finding no overall difference in muscle strength and function over 12 months. Two small RCTs (n = 52) compared daily prednisone 0.75 mg/kg/day with daily deflazacort 0.9 mg/kg/day, but it was not possible to compare muscle strength or function.Non-randomised studies support RCT evidence in showing improved functional benefit from corticosteroids. These studies suggest sustained benefit for up to 66 months.
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