Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status
Publication: Journal of Comparative Effectiveness Research
Abstract
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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Received: 2 November 2022
Accepted: 20 January 2023
Published online: 7 February 2023
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Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status. (2023) Journal of Comparative Effectiveness Research. DOI: 10.57264/cer-2022-0190
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