Steroid switching in dystrophinopathy treatment: a US chart review of patient characteristics and clinical outcomes
Journal of Comparative Effectiveness Research, 2021
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.
Authors
Marden JR, Santos C, Pfister B, Able R, Lane H, Somma M, Zhao J, Signorovitch J, Parsons J, Apkon S