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dc.contributor.authorMcDonald, Craig M
dc.contributor.authorMarden, Jessica R
dc.contributor.authorShieh, Perry B
dc.contributor.authorWong, Brenda L
dc.contributor.authorLane, Henry
dc.contributor.authorZhang, Adina
dc.contributor.authorNguyen, Ha
dc.contributor.authorFrean, Molly
dc.contributor.authorTrifillis, Panayiota
dc.contributor.authorKoladicz, Karyn
dc.contributor.authorSignorovitch, James
dc.date.accessioned2023-06-02T00:27:22Z
dc.date.available2023-06-02T00:27:22Z
dc.date.issued2023-02-07
dc.identifier.citationMcDonald CM, Marden JR, Shieh PB, Wong BL, Lane H, Zhang A, Nguyen H, Frean M, Trifillis P, Koladicz K, Signorovitch J. Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status. J Comp Eff Res. 2023 Apr;12(4):e220190. doi: 10.57264/cer-2022-0190. Epub 2023 Feb 7. PMID: 36749302.en_US
dc.identifier.eissn2042-6313
dc.identifier.doi10.57264/cer-2022-0190en_US
dc.identifier.pmid36749302
dc.identifier.urihttp://hdl.handle.net/20.500.14038/52135
dc.description.abstractAim: To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease progression. Methods: Data from daily steroid users (placebo-treated) were pooled from four phase 2b/3 trials in DMD. Outcomes assessed overall and among subgroups included changes from baseline to 48 weeks in six-minute walk distance (6MWD), timed function tests and North Star Ambulatory Assessment total score. Results: Among 231 patients receiving deflazacort (n = 127) or prednisone (n = 104), observed differences in 6MWD favoring deflazacort over prednisone were significant for patients with relatively older age (≥8-years-old), greater disease progression (baseline timed stand from supine ≥5 s), or longer corticosteroid use (>3 years). Conclusion: Daily deflazacort had greater benefits than daily prednisone particularly among older/more progressed patients.en_US
dc.language.isoenen_US
dc.relation.ispartofJournal of Comparative Effectiveness Researchen_US
dc.relation.urlhttps://doi.org/10.57264/cer-2022-0190en_US
dc.rightsOpen access: This work is licensed under the Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license,visit http://creativecommons.org/licenses/by-nc-nd/4.0/en_US
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 International*
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/*
dc.subjectDuchenne muscular dystrophyen_US
dc.subjectclinical outcomesen_US
dc.subjectdeflazacorten_US
dc.subjectdisease milestonesen_US
dc.subjectefficacyen_US
dc.subjectprednisone/prednisoloneen_US
dc.titleDisease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional statusen_US
dc.typeJournal Articleen_US
dc.source.journaltitleJournal of comparative effectiveness research
dc.source.volume12
dc.source.issue4
dc.source.beginpagee220190
dc.source.endpage
dc.source.countryEngland
dc.identifier.journalJournal of comparative effectiveness research
refterms.dateFOA2023-06-02T00:27:23Z
dc.contributor.departmentPediatricsen_US


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Open access: This work is licensed under the Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license,visit http://creativecommons.org/licenses/by-nc-nd/4.0/
Except where otherwise noted, this item's license is described as Open access: This work is licensed under the Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license,visit http://creativecommons.org/licenses/by-nc-nd/4.0/