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

<p dir="ltr"><b>These are peer-reviewed supplementary tables for the article '</b><b>Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy</b><b>' published in the</b><b> </b><b><i>Journal of Comparative Effectiveness Research</i></b><b>.</b></p><ul><li><b>Supplementary Table 1.</b> Search strategy for Ovid MEDLINE® (1 January 2000 to 30 November 2020)</li><li><b>Supplementary Table 2.</b> Search strategy for Ovid EMBASE® (1 January 2000 to 30 November 2020)</li><li><b>Supplementary Table 3.</b> Primary and post-hoc studies of patients receiving deflazacort or prednisone/prednisolone.</li></ul><p dir="ltr"><b>Summary: </b><b>Aim:</b> 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. <b>Materials & methods:</b> 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). <b>Results:</b> 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. <b>Conclusion:</b> 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.</p>