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

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.