Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

• Main Authors: Craig M McDonald (Author), Jessica R Marden (Author), Perry B Shieh (Author), Brenda L Wong (Author), Henry Lane (Author), Adina Zhang (Author), Ha Nguyen (Author), Molly Frean (Author), Panayiota Trifillis (Author), Karyn Koladicz (Author), James Signorovitch (Author)
• Format: Book
• Published: Becaris Publishing Limited, 2023-02-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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.

Item Description

10.57264/cer-2022-0190
2042-6313