Investigators at the Dubowitz Neuromuscular Centre, Great Ormond Street Hospital, and other centers in the UK, conducted a prospective longitudinal study across 17 neuromuscular centers in the UK of 360 boys aged 3-15 years with Duchenne muscular dystrophy who were treated with daily or intermittent (10 days on/10 days off) prednisolone for a mean duration of 4 years. The median loss of ambulation was 12 years in intermittent and 14.5 years in daily treatment; height restriction for intermittent versus daily regimen was 1.57 (p=0.13) and the median age for loss of ambulation did not differ. Boys on an intermittent regimen declined faster than those receiving daily treatment (p<0.001). Moderate to severe side effects were more common in the daily regimen, including Cushingoid features, hyperactive behavior and hypertension. Body mass index mean score was higher and height restriction was more severe in the daily regimen than in the intermittent regimen. [1]

COMMENT. Glucocorticoids are recommended in the international standards of care guideline for DMD and benefits are confirmed by Cochrane systematic reviews [2]. The most effective treatment postulated is prednisolone/prednisone or the equivalent deflazacort [3]. The intermittent and daily regimens are equally effective in gain of function until 6 years of age. After age 7 years, boys on an intermittent regimen decline more rapidly than those on daily therapy. Side effects are a greater problem with daily compared to intermittent therapy.