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

These are peer-reviewed supplementary tables for the article 'Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy' published in the Journal of Comparative Effectiveness Research.Supplementary Table 1. Search strategy for Ovid MEDLINE® (1 January 2000 to 30 November 2020)Supplementary Table 2. Search strategy for Ovid EMBASE® (1 January 2000 to 30 November 2020)Supplementary Table 3. Primary and post-hoc studies of patients receiving deflazacort or prednisone/prednisolone.Summary: 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.

本数据集为发表于《比较疗效研究杂志》之论文《deflazacort 与 prednisone/prednisolone 在无义突变型杜氏肌营养不良症患者的Meta分析》的同行评审补充表格。补充表格1：Ovid MEDLINE®（2000年1月1日至2020年11月30日）的检索策略；补充表格2：Ovid EMBASE®（2000年1月1日至2020年11月30日）的检索策略；补充表格3：接受deflazacort或prednisone/prednisolone治疗的患者的初步及事后研究。摘要：研究目的：比较deflazacort与prednisone/prednisolone在为无义突变型杜氏肌营养不良症患者提供临床上有意义的运动里程碑丧失延缓方面的疗效。材料与方法：将来自IIb期临床试验（ClinicalTrials.gov识别号：NCT00592553）和ACT DMD临床试验（ClinicalTrials.gov识别号：NCT01826487）的安慰剂数据在Meta分析中回顾性合并（意向治疗人群；针对基线至第48周6分钟步行距离[6MWD]及计时功能测试的变化）。结果：与prednisone/prednisolone相比，deflazacort在6分钟步行距离的变化上显示出显著改善（均方差差值39.54米[95%CI：13.799，65.286；p = 0.0026]）。与prednisone/prednisolone相比，deflazacort在4级楼梯爬升和4级楼梯下降方面显示出显著且具有临床意义的改善。结论：与prednisone/prednisolone相比，deflazacort在48周内为无义突变型杜氏肌营养不良症患者提供了临床上有意义的运动里程碑丧失延缓。