{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,6]],"date-time":"2026-11-06T03:23:41Z","timestamp":1793935421754,"version":"4.8.0"},"reference-count":38,"publisher":"SAGE Publications","issue":"1","license":[{"start":{"date-parts":[[2022,12,19]],"date-time":"2022-12-19T00:00:00Z","timestamp":1671408000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2023,1,3]]},"abstract":"<jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Evidence on the long-term efficacy of steroids in Duchenne muscular dystrophy (DMD) after loss of ambulation is limited.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>Characterize and compare disease progression by steroid treatment (prednisone, deflazacort, or no steroids) among non-ambulatory boys with DMD.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>Disease progression was measured by functional status (Performance of Upper Limb Module for DMD 1.2 [PUL] and Egen Klassifikation Scale Version 2 [EK] scale) and by cardiac and pulmonary function (left ventricular ejection fraction [LVEF], forced vital capacity [FVC] % -predicted, cough peak flow [CPF]). Longitudinal changes in outcomes, progression to key disease milestones, and dosing and body composition metrics were analyzed descriptively and in multivariate models.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>\n                      This longitudinal cohort study included 86 non-ambulatory patients with DMD (mean age 13.4 years;\n                      <jats:italic>n<\/jats:italic>\n                      \u200a=\u200a40 [deflazacort],\n                      <jats:italic>n<\/jats:italic>\n                      \u200a=\u200a29 [prednisone],\n                      <jats:italic>n<\/jats:italic>\n                      \u200a=\u200a17 [no steroids]). Deflazacort use resulted in slower average declines in FVC % -predicted vs. no steroids (+3.73 percentage points\/year,\n                      <jats:italic>p<\/jats:italic>\n                      \u200a&lt;\u200a0.05). Both steroids were associated with significantly slower average declines in LVEF, improvement in CPF, and slower declines in total PUL score and EK total score vs. no steroids; deflazacort was associated with slower declines in total PUL score vs. prednisone (all\n                      <jats:italic>p<\/jats:italic>\n                      \u200a&lt;\u200a0.05). Both steroids also preserved functional abilities considered especially important to quality of life, including the abilities to perform hand-to-mouth function and to turn in bed at night unaided (all\n                      <jats:italic>p<\/jats:italic>\n                      \u200a&lt;\u200a0.05 vs. no steroids).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec specific-use=\"heading-level-1\">\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>Steroid use after loss of ambulation in DMD was associated with delayed progression of important pulmonary, cardiac, and upper extremity functional deficits, suggesting some benefits of deflazacort over prednisone.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-221575","type":"journal-article","created":{"date-parts":[[2022,12,20]],"date-time":"2022-12-20T12:58:27Z","timestamp":1671541107000},"page":"67-79","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":19,"title":["Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy"],"prefix":"10.1177","volume":"10","author":[{"given":"Craig M.","family":"McDonald","sequence":"first","affiliation":[{"name":"University of California Davis Health, Sacramento, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Oscar H.","family":"Mayer","sequence":"additional","affiliation":[{"name":"Children\u2019s Hospital of Philadelphia, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kan N.","family":"Hor","sequence":"additional","affiliation":[{"name":"Nationwide Children\u2019s Hospital, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Debra","family":"Miller","sequence":"additional","affiliation":[{"name":"CureDuchenne, Newport Beach, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathalie","family":"Goemans","sequence":"additional","affiliation":[{"name":"University of Leuven, Leuven, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erik K.","family":"Henricson","sequence":"additional","affiliation":[{"name":"University of California Davis Health, Sacramento, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica R.","family":"Marden","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan","family":"Freimark","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henry","family":"Lane","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adina","family":"Zhang","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Molly","family":"Frean","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Panayiota","family":"Trifillis","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc. 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South Plainfield, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"Signorovitch","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"on behalf of the PRO-DMD-01 consortium investigators","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2022,12,19]]},"reference":[{"key":"e_1_3_4_2_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24880"},{"key":"e_1_3_4_3_2","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2014-2044"},{"key":"e_1_3_4_4_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23528"},{"key":"e_1_3_4_5_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01430-8"},{"key":"e_1_3_4_6_2","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2012-303902"},{"key":"e_1_3_4_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2015.07.009"},{"key":"e_1_3_4_8_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2013.06.001"},{"key":"e_1_3_4_9_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0113999"},{"key":"e_1_3_4_10_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2010.09.006"},{"key":"e_1_3_4_11_2","first-page":"1475","article-title":"Assessment and management of respiratory function in patients with Duchenne muscular dystrophy: Current and emerging options","volume":"11","author":"LoMauro A","year":"2015","unstructured":"LoMauroA, D\u2019AngeloMG, AlivertiAAssessment and management of respiratory function in patients with Duchenne muscular dystrophy: Current and emerging options. 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