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dc.contributor.authorMarden, Jessica R.
dc.contributor.authorFreimark, Jonathan
dc.contributor.authorYao, Zhiwen
dc.contributor.authorSignorovitch, James
dc.contributor.authorTian, Cuixia
dc.contributor.authorWong, Brenda L
dc.date2022-08-11T08:09:55.000
dc.date.accessioned2022-08-23T16:48:48Z
dc.date.available2022-08-23T16:48:48Z
dc.date.issued2020-02-01
dc.date.submitted2020-02-20
dc.identifier.citation<p>Marden JR, Freimark J, Yao Z, Signorovitch J, Tian C, Wong BL. Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center. J Comp Eff Res. 2020 Feb;9(3):177-189. doi: 10.2217/cer-2019-0170. Epub 2020 Jan 10. PMID: 31922454. <a href="https://doi.org/10.2217/cer-2019-0170">Link to article on publisher's site</a></p>
dc.identifier.issn2042-6305 (Linking)
dc.identifier.doi10.2217/cer-2019-0170
dc.identifier.pmid31922454
dc.identifier.urihttp://hdl.handle.net/20.500.14038/41339
dc.description.abstractAim: To assess outcomes among patients with Duchenne muscular dystrophy receiving deflazacort or prednisone in real-world practice. Methods: Clinical data for 435 boys with Duchenne muscular dystrophy from Cincinnati Children's Hospital Medical Center were studied retrospectively using time-to-event and regression analyses. Results: Median ages at loss of ambulation were 15.6 and 13.5 years among deflazacort- and prednisone-initiated patients, respectively. Deflazacort was also associated with a lower risk of scoliosis and better ambulatory function, greater % lean body mass, shorter stature and lower weight, after adjusting for age and steroid duration. No differences were observed in whole body bone mineral density or left ventricular ejection fraction. Conclusion: This single center study adds to the real-world evidence associating deflazacort with improved clinical outcomes.
dc.language.isoen_US
dc.relation<p><a href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&list_uids=31922454&dopt=Abstract">Link to Article in PubMed</a></p>
dc.rights© 2020 Analysis Group, Inc. 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/
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
dc.subjectDuchenne muscular dystrophy
dc.subjectdeflazacort
dc.subjectprednisone
dc.subjectreal-world data
dc.subjectCongenital, Hereditary, and Neonatal Diseases and Abnormalities
dc.subjectHealth Services Administration
dc.subjectHealth Services Research
dc.subjectMusculoskeletal Diseases
dc.subjectNervous System Diseases
dc.subjectPediatrics
dc.subjectTherapeutics
dc.titleReal-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center
dc.typeJournal Article
dc.source.journaltitleJournal of comparative effectiveness research
dc.source.volume9
dc.source.issue3
dc.identifier.legacyfulltexthttps://escholarship.umassmed.edu/cgi/viewcontent.cgi?article=5142&amp;context=oapubs&amp;unstamped=1
dc.identifier.legacycoverpagehttps://escholarship.umassmed.edu/oapubs/4123
dc.identifier.contextkey16607008
refterms.dateFOA2022-08-23T16:48:49Z
html.description.abstract<p>Aim: To assess outcomes among patients with Duchenne muscular dystrophy receiving deflazacort or prednisone in real-world practice.</p> <p>Methods: Clinical data for 435 boys with Duchenne muscular dystrophy from Cincinnati Children's Hospital Medical Center were studied retrospectively using time-to-event and regression analyses.</p> <p>Results: Median ages at loss of ambulation were 15.6 and 13.5 years among deflazacort- and prednisone-initiated patients, respectively. Deflazacort was also associated with a lower risk of scoliosis and better ambulatory function, greater % lean body mass, shorter stature and lower weight, after adjusting for age and steroid duration. No differences were observed in whole body bone mineral density or left ventricular ejection fraction.</p> <p>Conclusion: This single center study adds to the real-world evidence associating deflazacort with improved clinical outcomes.</p>
dc.identifier.submissionpathoapubs/4123
dc.contributor.departmentDepartment of Pediatrics
dc.source.pages177-189


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© 2020 Analysis Group, Inc. 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 © 2020 Analysis Group, Inc. 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/