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

• Published in: Journal of comparative effectiveness research 2021-12, Vol.10 (18), p.1337-1347
• Main Authors: Shieh, Perry B, Elfring, Gary, Trifillis, Panayiota, Santos, Claudio, Peltz, Stuart W, Parsons, Julie A, Apkon, Susan, Darras, Basil T, Campbell, Craig, McDonald, Craig M
• Format: Article
• Language: English
• Subjects: Codon, Nonsense; deflazacort; Duchenne muscular dystrophy; Humans; meta-analysis; Muscular Dystrophy, Duchenne - drug therapy; Muscular Dystrophy, Duchenne - genetics; nonsense mutation Duchenne muscular dystrophy; prednisolone; Prednisolone - therapeutic use; prednisone; Prednisone - therapeutic use; Pregnenediones; Retrospective Studies
• ISSN: 2042-6305; 2042-6313
• DOI: 10.2217/cer-2021-0018

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. 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). 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. 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.