Steroid switching in dystrophinopathy treatment: a US chart review of patient characteristics and clinical outcomes
| Autor: | Henry Lane, James Signorovitch, Michael Somma, Jessica R. Marden, Susan D. Apkon, Claudio Santos, Julie Parsons, Jing Zhao, Richard Able, Brian Pfister |
|---|---|
| Rok vydání: | 2021 |
| Předmět: |
musculoskeletal diseases
Pediatrics medicine.medical_specialty business.industry Health Policy Duchenne muscular dystrophy medicine.disease Muscular Dystrophy Duchenne Deflazacort 03 medical and health sciences 0302 clinical medicine Tolerability Prednisone 030225 pediatrics Chart review medicine Prednisolone Humans Muscular dystrophy business Adverse effect 030217 neurology & neurosurgery medicine.drug |
| Zdroj: | Journal of Comparative Effectiveness Research. 10:1065-1078 |
| ISSN: | 2042-6313 2042-6305 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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