Balancing Benefits and Risks: Long-term Use of Daily versus Intermittent Corticosteroids in Duchenne Muscular Dystrophy

BACKGROUND: Duchenne muscular dystrophy (DMD) is treated with intermittent or daily corticosteroid (CS) regimens. Risk-benefit assessment is essential to optimise treatment choice, but long-term efficacy and side effects accumulate beyond clinical trial duration. We describe long-term effects of intermittent compared with daily regimens using real-world data. METHODS: We compared retrospective data from individuals with DMD treated daily with deflazacort versus intermittently (10 days on/off) with prednisone or deflazacort at three neuromuscular centres in Belgium and the Netherlands between 1995 and 2022. Outcomes included motor, pulmonary, cardiac, anthropometric and bone health domains, analysed using linear mixed effects models and beta regression. RESULTS: Data included 75 patients treated daily and 144 treated intermittently (median age start CS 5.6 years vs 5.5 years, mean follow-up 10.7 years vs 9.5 years). Daily treatment showed later loss of ambulation (15.0 years vs 11.0 years, p