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

dataset

posted on 2024-05-02, 14:09 authored by Jessica Marden, Claudio Santos, Brian Pfister, Richard Able, Henry Lane, Michael Somma, Jing Zhao, James Signorovitch, Julie Parsons, Susan Apkon

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 methods
Supplemental Table 1: Data availability

Aim: 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.