Intermittent versus daily regimen of prednisolone in ambulatory boys with Duchenne muscular dystrophy: A randomized, open-label trial.

Introduction/aims: Corticosteroids prolong ambulation and improve muscle power among boys with Duchenne muscular dystrophy (DMD). However, the optimal steroid regimen remains unclear. Hence, this study was undertaken to compare the efficacy of daily- versus intermittent-steroid regimens in ambulatory boys with DMD.

Methods: In this single-center, open-label randomized trial, 72 children were randomized to receive either daily prednisolone (0.75 mg/kg/day) or intermittent prednisolone (0.75 mg/kg/day, for first 10 days of every month). The primary outcome measure was the difference in average score on manual muscle testing (MMT) at baseline and after 6 mo of steroids. A difference of >0.2 was hypothesized to be significant. Secondary outcomes included changes in timed functions, muscular dystrophy-specific functional-rating scale score, peak torque, average power, and pulmonary function.

Results: In the intention-to-treat analysis, the mean (SD) change in MMT scores was 0.17 (0.15) and 0.08 (0.10) for the daily and intermittent steroid groups, respectively. The mean difference between the two interventions was 0.10 (95% confidence interval [CI] = 0.04-0.16; P = .003), which although significant was less than the predefined value of 0.2. Statistically significantly improvements were observed with daily-steroid regimen in the Gowers time (P = .01), nine-metre walk test (P = .02) and average power (P = .02) as compared to intermittent-steroid regimen. A total of 19/32 (52.8%) children in the daily-steroid group and 8/29 (27%) children in the intermittent-steroid group experienced some form of adverse effect (P = .02).

Discussion: Over a short-term period, the intermittent-steroid regimen was non-inferior to the daily-steroid regime in preserving muscle strength among children with DMD. However, better improvement of functional measures was observed with daily-steroid administration. The frequency of individual side effects was similar between the two groups.