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.