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.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2021 |
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| Erschienen: |
2021 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:10 |
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| Enthalten in: |
Journal of comparative effectiveness research - 10(2021), 14 vom: 03. Okt., Seite 1065-1078 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Marden, Jessica R [VerfasserIn] |
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| Links: |
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| Themen: |
Becker muscular dystrophy |
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| Anmerkungen: |
Date Completed 27.10.2021 Date Revised 27.10.2021 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.2217/cer-2021-0110 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM328206946 |
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| 245 | 1 | 0 | |a Steroid switching in dystrophinopathy treatment |b a US chart review of patient characteristics and clinical outcomes |
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| 500 | |a Citation Status MEDLINE | ||
| 520 | |a 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 | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a Becker muscular dystrophy | |
| 650 | 4 | |a Duchenne muscular dystrophy | |
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| 700 | 1 | |a Lane, Henry |e verfasserin |4 aut | |
| 700 | 1 | |a Somma, Michael |e verfasserin |4 aut | |
| 700 | 1 | |a Zhao, Jing |e verfasserin |4 aut | |
| 700 | 1 | |a Signorovitch, James |e verfasserin |4 aut | |
| 700 | 1 | |a Parsons, Julie |e verfasserin |4 aut | |
| 700 | 1 | |a Apkon, Susan |e verfasserin |4 aut | |
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