{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,20]],"date-time":"2026-09-20T12:17:12Z","timestamp":1789906632796,"version":"4.0.1"},"reference-count":29,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2020,11,24]],"date-time":"2020-11-24T00:00:00Z","timestamp":1606176000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006396","name":"Alexion Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006396","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Neurol."],"abstract":"<jats:p><jats:bold>Introduction:<\/jats:bold>Chronic, broad-spectrum immunosuppressive therapy (IST) can be associated with side effects in many people with generalized myasthenia gravis (gMG), and treatment guidelines recommend that the IST dose be tapered once patients achieve a stable treatment response. We therefore examined IST use in eculizumab-treated patients with refractory gMG.<\/jats:p><jats:p><jats:bold>Methods:<\/jats:bold>The REGAIN open-label extension (OLE) enrolled 117 adults with refractory anti-acetylcholine receptor antibody-positive gMG who had completed the 6-month, randomized, double-blind, placebo-controlled REGAIN study of eculizumab. Eligible patients had received \u22652 ISTs for \u22651 year or \u22651 IST with intravenous immunoglobulin or plasma exchange \u22654 times in 1 year, without symptom control. During REGAIN, changes in concomitant MG therapies were not permitted; during the OLE, they were permitted at the investigators' discretion. Participants received eculizumab 1,200 mg every 2 weeks for up to 4 years; concomitant prednisone and related corticosteroids (PRED), azathioprine (AZA), and mycophenolate mofetil (MMF) use was recorded. Changes in MG Activities of Daily Living and Quantitative MG total scores, MG exacerbations, and adverse events were also recorded.<\/jats:p><jats:p><jats:bold>Results:<\/jats:bold>At last OLE assessment, 88.0% (103\/117) of participants were using \u22651 IST vs. 98.3% (115\/117) at OLE baseline. During the OLE, 76.9% (90\/117) of patients experienced a total of 719 IST changes. Almost half of participants [48.7% (57\/117)] stopped or decreased \u22651 IST owing to MG symptom improvement, representing 38.9% (280\/719) of all changes. In patients who decreased and\/or stopped \u22651 IST, mean daily doses of PRED, AZA, and MMF decreased between OLE baseline and last assessment by 60.8% [standard deviation (SD), 28.07;<jats:italic>P<\/jats:italic>&amp;lt; 0.0001], 89.1% (SD, 25.77;<jats:italic>P<\/jats:italic>&amp;lt; 0.0001), and 56.0% (SD, 32.99;<jats:italic>P<\/jats:italic>&amp;lt; 0.0001), respectively. Improved clinical outcomes were observed with eculizumab regardless of IST changes during the OLE, and eculizumab's safety profile was similar in patients who used PRED, AZA, and MMF.<\/jats:p><jats:p><jats:bold>Conclusions:<\/jats:bold>Use of ISTs by patients with previously refractory gMG decreased during eculizumab treatment in the REGAIN OLE. Clinical improvements with eculizumab were maintained by patients in all groups, including those who decreased and\/or stopped concomitant ISTs.<\/jats:p><jats:p><jats:bold>Trial registration:<\/jats:bold><jats:ext-link>www.clinicaltrials.gov<\/jats:ext-link>: NCT01997229, NCT02301624.<\/jats:p>","DOI":"10.3389\/fneur.2020.556104","type":"journal-article","created":{"date-parts":[[2020,11,24]],"date-time":"2020-11-24T05:54:53Z","timestamp":1606197293000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":34,"title":["Concomitant Immunosuppressive Therapy Use in Eculizumab-Treated Adults With Generalized Myasthenia Gravis During the REGAIN Open-Label Extension Study"],"prefix":"10.3389","volume":"11","author":[{"given":"Richard J.","family":"Nowak","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Srikanth","family":"Muppidi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Said R.","family":"Beydoun","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fanny L.","family":"O'Brien","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcus","family":"Yountz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"Jr.","given":"James F.","family":"Howard","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2020,11,24]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"1054","DOI":"10.1212\/WNL.26.11.1054","article-title":"Antibody to acetylcholine receptor in myasthenia gravis. 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