Disease Progression Rates in Ambulatory Duchenne Muscular Dystrophy by Steroid Type, Patient Age and Functional Status
Journal of Comparative Effectiveness Research, 2023
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.
Authors
McDonald CM, Marden JR, Shieh PB, Wong BL, Lane H, Zhang A, Nguyen H, Frean M, Trifillis P, Koladicz K, Signorovitch J