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

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.

• Authors: Perry B Shieh, Gary Elfring, Panayiota Trifillis, Claudio Santos, Stuart W Peltz, Julie A Parsons, Susan Apkon, Basil T Darras, Craig Campbell, Craig M McDonald, Members of the Ataluren Phase IIb Study Group, Members of the Ataluren Phase IIb Study Clinical Evaluator Training Group, Members of the ACT DMD Study Group, Members of the ACT DMD Clinical Evaluator Training Group
• Journal: Journal of comparative effectiveness research (J Comp Eff Res) Vol. 10 Issue 18 Pg. 1337-1347 (12 2021) ISSN: 2042-6313 [Electronic] England
• PMID: 34693725 (Publication Type: Journal Article, Meta-Analysis, Research Support, Non-U.S. Gov't)

Chemical References

• Codon, Nonsense
• Pregnenediones
• Prednisolone
• deflazacort
• Prednisone

Topics

• Codon, Nonsense
• Humans
• Muscular Dystrophy, Duchenne (drug therapy, genetics)
• Prednisolone (therapeutic use)
• Prednisone (therapeutic use)
• Pregnenediones
• Retrospective Studies