Open AccessSteroid switching in dystrophinopathy treatment: a US chart review of patient characteristics and clinical outcomes
Author(s) -
Jessica R. Marden,
Claudio Santos,
Brian Pfister,
Richard Able,
H. Clifford Lane,
Michael Somma,
Jing Zhao,
James Signorovitch,
Julie Parsons,
Susan Apkon
Publication year - 2021
Publication title -
journal of comparative effectiveness research
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.567
H-Index - 23
eISSN - 2042-6313
pISSN - 2042-6305
DOI - 10.2217/cer-2021-0110
Subject(s) - deflazacort , medicine , prednisone , duchenne muscular dystrophy , prednisolone , muscular dystrophy , tolerability , physical therapy , pediatrics , adverse effect
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.