期刊
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH
卷 9, 期 14, 页码 973-984出版社
Becaris Publishing
DOI: 10.2217/cer-2020-0095
关键词
6-minute walk distance; ataluren; Duchenne muscular dystrophy; efficacy; meta-analyses; nonsense mutation Duchenne muscular dystrophy; randomized controlled trials
资金
- PTC Therapeutics
- Fondazione Telethon
- Italian Ministry of Health
- National Institutes of Health/National Institute of Neurological Disorders
- Slaney Fund for SMA
- SMA Foundation
- Stroke
- AveXis
- Biogen
- Cytokinetics
- Fibrogen
- Ionis Pharmaceuticals
- Santhera Pharmaceuticals
- Summit Therapeutics
- Muscular Dystrophy Association
- National Institutes of Health
- BioMarin
- Bristol-Myers Squibb
- Catabasis
- Eli Lilly
- Mitobridge
- Novartis
- ReveraGen
- Roche
- Sarepta Therapeutics
- Beauhawks Foundation
- CureDuchenne
- NIHR Great Ormond Street Hospital Biomedical Research Centre
- Esperare
- Pfizer
- Fundacion Isabel Gemio
- Instituto de Salud Carlos III
- Orphazyme
- Patient-Centered Outcomes Research Institute (PCORI)
- Ra Pharma
- Sanofi Genzyme
- US FDA Office of Orphan Products Development
- ReveraGen BioPharma
- NS Pharma
- Italfarmaco
- Genzyme
Aim:Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD).Materials & methods:Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT) populations and two patient subgroups (baseline 6-min walk distance [6MWD] >= 300-<400 or <400 m). Meta-analyses examined 6MWD change from baseline to week 48.Results:Statistically significant differences in 6MWD change with ataluren versus placebo were observed across all three meta-analyses. Least-squares mean difference (95% CI): ITT (n = 342), +17.2 (0.2-34.1) m, p = 0.0473; >= 300-<400 m (n = 143), +43.9 (18.2-69.6) m, p = 0.0008; <400 m (n = 216), +27.7 (6.4-49.0) m, p = 0.0109.Conclusion:These meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMD over 48 weeks. Treatment benefit was most evident in patients with a baseline 6MWD >= 300-<400 m (the ambulatory transition phase), thereby informing future trial design.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据