Steroid switching in dystrophinopathy treatment: a US chart review of patient characteristics and clinical outcomes.
Jessica R MardenClaudio SantosBrian PfisterRichard AbleHenry LaneMichael SommaJing ZhaoJames SignorovitchJulie ParsonsSusan ApkonPublished in: 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.
Keyphrases
- muscular dystrophy
- duchenne muscular dystrophy
- end stage renal disease
- emergency department
- chronic kidney disease
- ejection fraction
- newly diagnosed
- primary care
- prognostic factors
- adipose tissue
- type diabetes
- clinical trial
- metabolic syndrome
- patient reported outcomes
- insulin resistance
- combination therapy
- weight loss
- skeletal muscle