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Res."],"published-print":{"date-parts":[[2020,2]]},"abstract":"<jats:p>\n                    <jats:bold>Aim:<\/jats:bold>\n                    To assess outcomes among patients with Duchenne muscular dystrophy receiving deflazacort or prednisone in real-world practice.\n                    <jats:bold>Methods:<\/jats:bold>\n                    Clinical data for 435 boys with Duchenne muscular dystrophy from Cincinnati Children\u2019s Hospital Medical Center were studied retrospectively using time-to-event and regression analyses.\n                    <jats:bold>Results:<\/jats:bold>\n                    Median ages at loss of ambulation were 15.6 and 13.5\u00a0years 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.\n                    <jats:bold>Conclusion:<\/jats:bold>\n                    This single center study adds to the real-world evidence associating deflazacort with improved clinical outcomes.\n                  <\/jats:p>","DOI":"10.2217\/cer-2019-0170","type":"journal-article","created":{"date-parts":[[2020,1,10]],"date-time":"2020-01-10T10:28:29Z","timestamp":1578652109000},"page":"177-189","source":"Crossref","is-referenced-by-count":47,"title":["Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center"],"prefix":"10.57264","volume":"9","author":[{"given":"Jessica R","family":"Marden","sequence":"first","affiliation":[{"name":"Analysis Group, Inc., Boston, MA 02199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"Freimark","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA 02199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhiwen","family":"Yao","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA 02199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"Signorovitch","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA 02199, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cuixia","family":"Tian","sequence":"additional","affiliation":[{"name":"Cincinnati Children\u2019s Hospital Medical Center &amp; Department of Pediatrics, College of Medicine, University of Cincinnati, Cincinnati, OH 45229, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brenda L","family":"Wong","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01655, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","reference":[{"key":"e_1_3_7_3_2","unstructured":"Genetic and Rare Diseases Information Center. 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