Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy

Authors

Perry B. Shieh, University of California, Los Angeles
Gary Elfring, PTC Therapeutics
Panayiota Trifillis, PTC Therapeutics
Claudio Santos, PTC Therapeutics
Stuart W. Peltz, PTC Therapeutics
Julie A. Parsons, University of Colorado School of Medicine
Susan Apkon, University of Colorado School of Medicine
Basil T. Darras, Children's Hospital Boston

Document Type

Article

Publication Date

12-1-2021

Journal

Journal of Comparative Effectiveness Research

Volume

10

Issue

18

First Page

1337

Last Page

1347

URL with Digital Object Identifier

10.2217/cer-2021-0018

Abstract

Aim: Compare efficacies of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy. Materials & methods: Placebo data from Phase IIb (ClinicalTrials.gov Identifier: NCT00592553) and ACT DMD (ClinicalTrials.gov Identifier: NCT01826487) ataluren nonsense mutation Duchenne muscular dystrophy clinical trials were retrospectively combined in meta-analyses (intent-to-treat population; for change from baseline to week 48 in 6-min walk distance [6MWD] and timed function tests). Results: Significant improvements in change in 6-min walk distance with deflazacort versus prednisone/prednisolone (least-squares mean difference 39.54 m [95% CI: 13.799, 65.286; p = 0.0026]). Significant and clinically meaningful improvements in 4-stair climb and 4-stair descend for deflazacort versus prednisone/prednisolone. Conclusion: Deflazacort provides clinically meaningful delays in loss of physical milestones over 48 weeks compared with prednisone/prednisolone for patients with nonsense mutation Duchenne muscular dystrophy.