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

<b>These are peer-reviewed supplementary materials for the article '</b><b>Steroid switching in dystrophinopathy </b><b>treatment: a US chart review of patient </b><b>characteristics and clinical outcomes</b><b>' published in the</b><b> </b><b><i>Journal of Comparative Effectiveness Research</i></b><b>.</b><b>Supplementary methods</b><b>Supplemental Table 1: </b>Data availability<b>Aim:</b> 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. <b>Methods: </b>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. <b>Results:</b> 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. <b>Conclusion:</b> Physician-reported outcomes were consistent with deflazacort addressing patients’ primary reasons for switching.

本文为发表于《比较效果研究杂志》（Journal of Comparative Effectiveness Research）的文章《肌营养不良症的类固醇换药治疗：美国患者特征与临床结局的病历回顾研究》的经同行评议补充材料。

### 补充方法
#### 补充表1：数据可用性
**研究目的**：描述美国杜氏肌营养不良症与贝克肌营养不良症（Duchenne and Becker muscular dystrophy，DMD与BMD）患者从泼尼松（prednisone）/泼尼松龙（prednisolone）换用去氟可特（deflazacort）的原因，及其对应的相关临床结局。
**研究方法**：对2017年2月至2018年12月期间完成泼尼松至去氟可特换药的62例DMD患者与30例BMD患者开展病历回顾研究，收集其人口统计学与临床特征、换药原因、治疗结局及常见不良事件。
**研究结果**：患者换药时的平均年龄分别为DMD组20.1岁、BMD组9.2岁。医师报告的主要换药原因为「延缓疾病进展」（DMD组占比83%，BMD组占比79%）与「药物耐受性」（DMD组占比67%，BMD组占比47%）。90%~95%的患者中，换药在解决主要换药原因方面被评为「非常有效」或「较为有效」。
**研究结论**：医师报告的治疗结局与去氟可特能够匹配患者换药的核心需求相符。