| Summary: | Aim: To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease
progression. Methods: Data from daily steroid users (placebo-treated) were pooled from four phase
2b/3 trials in DMD. Outcomes assessed overall and among subgroups included changes from baseline to
48 weeks in six-minute walk distance (6MWD), timed function tests and North Star Ambulatory Assessment
total score. Results: Among 231 patients receiving deflazacort (n = 127) or prednisone (n = 104), observed
differences in 6MWD favoring deflazacort over prednisone were significant for patients with relatively
older age (≥8-years-old), greater disease progression (baseline timed stand from supine ≥5 s), or longer
corticosteroid use (>3 years). Conclusion: Daily deflazacort had greater benefits than daily prednisone
particularly among older/more progressed patients.
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