Supplemental materials: Steroid switching in dystrophinopathy treatment: a US chart review of patient characteristics and clinical outcomes

These are peer-reviewed supplementary materials for the article 'Steroid switching in dystrophinopathy treatment: a US chart review of patient characteristics and clinical outcomes' published in the Journal of Comparative Effectiveness Research.Supplementary methodsSupplemental Table 1: Data availabilityAim: To describe reasons for switching from prednisone/prednisolone to deflazacort and associated clinical outcomes among patients with Duchenne and Becker muscular dystrophy (DMD and BMD, respectively) in the USA. Methods: A chart review of patients with DMD (n = 62) or BMD (n = 30) who switched from prednisone to deflazacort (02/2017–12/2018) collected demographic/clinical characteristics, reasons for switching, outcomes and common adverse events. Results: The mean ages at switch were 20.1 (DMD) and 9.2 (BMD) years. The primary physician-reported reasons for switching were ‘to slow disease progression’ (DMD: 83%, BMD: 79%) and ‘tolerability’ (67 and 47%). Switching was ‘very’ or ‘somewhat’ effective at addressing the primary reasons in 90–95% of patients. Conclusion: Physician-reported outcomes were consistent with deflazacort addressing patients’ primary reasons for switching.

本数据集为发表于《比较疗效研究杂志》的论文《Duchenne 和 Becker 肌营养不良症治疗中的类固醇转换：美国病例回顾分析——患者特征与临床结果》的同行评审补充材料。补充方法：补充表 1：数据可用性目标：旨在描述从泼尼松/泼尼松龙转换为德法拉佐特的理由及其与美国杜氏肌营养不良症（DMD）和贝克肌营养不良症（BMD）患者相关的临床结果。方法：对 62 例 DMD 患者和 30 例 BMD 患者进行了病例回顾，这些患者在 2017 年 2 月至 2018 年 12 月期间从泼尼松转换为德法拉佐特，收集了人口统计学/临床特征、转换原因、结果和常见的不良事件。结果：转换时的平均年龄分别为 20.1 岁（DMD）和 9.2 岁（BMD）。主要医师报告的转换原因为‘减缓疾病进展’（DMD：83%，BMD：79%）和‘耐受性’（67% 和 47%）。90-95% 的患者中，转换对解决主要原因非常或有一定的效果。结论：医师报告的结果与德法拉佐特解决患者转换主要原因的观点一致。