关于无义突变型杜氏肌营养不良症患者使用地夫可特与泼尼松/强的松治疗的荟萃分析。
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy.
机构信息
University of California at Los Angeles, Los Angeles, CA, USA.
PTC Therapeutics, Inc., South Plainfield, NJ, USA.
出版信息
J Comp Eff Res. 2021 Dec;10(18):1337-1347. doi: 10.2217/cer-2021-0018. Epub 2021 Oct 25.
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.
比较地夫可特和泼尼松/强的松在治疗无义突变杜氏肌营养不良症患者丧失身体里程碑方面的疗效，以提供临床上有意义的延迟。来自 IIb 期（ClinicalTrials.gov 标识符：NCT00592553）和 ACT DMD（ClinicalTrials.gov 标识符：NCT01826487）阿替洛尔无义突变杜氏肌营养不良症临床试验的安慰剂数据在荟萃分析中进行了回顾性合并（意向治疗人群；6 分钟步行距离[6MWD]和计时功能测试从基线到第 48 周的变化）。与泼尼松/强的松相比，地夫可特在 6 分钟步行距离的变化方面有显著改善（最小二乘均数差异 39.54m[95%CI：13.799,65.286;p=0.0026]）。与泼尼松/强的松相比，地夫可特在 4 级爬梯和 4 级下梯测试中也有显著和有临床意义的改善。与泼尼松/强的松相比，地夫可特在 48 周内为无义突变杜氏肌营养不良症患者提供了临床上有意义的丧失身体里程碑的延迟。
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