{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T17:27:59Z","timestamp":1791912479786,"version":"4.3.4"},"reference-count":23,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2021,5,27]],"date-time":"2021-05-27T00:00:00Z","timestamp":1622073600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Ann Clin Transl Neurol"],"published-print":{"date-parts":[[2021,7]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>Generalized myasthenia gravis (gMG) is an autoimmune disease that causes disabling weakness via damage to the neuromuscular junction. In most patients, the disease is mediated by autoantibodies to the acetylcholine receptor, which activate the complement cascade. Our objective was to analyze response profiles in adult patients with anti\u2010acetylcholine receptor antibody\u2010positive refractory gMG treated with eculizumab\u2014a terminal complement inhibitor\u2014in the REGAIN study or its open\u2010label extension (OLE).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We retrospectively analyzed Myasthenia Gravis\u2010Activities of Daily Living (MG\u2010ADL) and Quantitative Myasthenia Gravis (QMG) scores recorded during REGAIN and its OLE. Early\/late responses were defined as improvement in MG\u2010ADL score (\u22653 points) or QMG score (\u22655 points) at \u226412 or &gt;12\u00a0weeks, respectively, after eculizumab initiation.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The analysis included 98 patients. By Week 12 and conclusion of the OLE, MG\u2010ADL response had been achieved at some point by 67.3% and 84.7% of patients, respectively, and QMG response by 56.1% and 71.4%, respectively. Response was observed over multiple consecutive assessments for most patients. At Week 130, the least\u2010squares mean percentage changes (95% CI) from baseline in MG\u2010ADL score were \u221261.9% (\u221269.9%, \u221253.9%) and \u221247.5% (\u221259.0%, \u221236.0%) in early and late MG\u2010ADL responders, respectively; the least\u2010squares mean percentage changes from baseline in QMG score were \u221240.8% (\u221248.3%, \u221233.4%) and \u221255.5% (\u221268.4%, \u221242.7%) in early and late QMG responders, respectively.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Interpretation<\/jats:title>\n                    <jats:p>The findings suggest that, although most patients with refractory gMG will achieve clinical response by Week 12 of eculizumab treatment, first responses can be observed with longer\u2010term treatment.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/acn3.51376","type":"journal-article","created":{"date-parts":[[2021,5,27]],"date-time":"2021-05-27T10:44:53Z","timestamp":1622112293000},"page":"1398-1407","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":64,"title":["Long\u2010term efficacy of eculizumab in refractory generalized myasthenia gravis: responder analyses"],"prefix":"10.1002","volume":"8","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7136-8617","authenticated-orcid":false,"suffix":"Jr","given":"James F.","family":"Howard","sequence":"first","affiliation":[{"name":"Department of Neurology The University of North Carolina Chapel Hill North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chafic","family":"Karam","sequence":"additional","affiliation":[{"name":"Department of Neurology Oregon Health &amp; Science University Portland Oregon USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcus","family":"Yountz","sequence":"additional","affiliation":[{"name":"Alexion Pharmaceuticals, Inc. Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fanny L.","family":"O\u2019Brien","sequence":"additional","affiliation":[{"name":"Alexion Pharmaceuticals, Inc. Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tahseen","family":"Mozaffar","sequence":"additional","affiliation":[{"name":"Department of Neurology University of California Irvine California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"for the REGAIN Study Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2021,5,27]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ncl.2018.01.002"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1097\/CND.0000000000000034"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1586\/eci.12.34"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.4049\/jimmunol.142.8.2654"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.4049\/jimmunol.140.8.2589"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0165-5728(96)00144-0"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1097\/00005072-198211000-00001"},{"key":"e_1_2_7_9_1","doi-asserted-by":"publisher","DOI":"10.1021\/bi00511a017"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.43.6.1167"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1097\/00005072-198003000-00005"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.1177\/1756285617749134"},{"key":"e_1_2_7_13_1","first-page":"255","article-title":"Clinical characteristics of refractory myasthenia gravis patients","volume":"86","author":"Suh J","year":"2013","journal-title":"Yale J Biol Med"},{"key":"e_1_2_7_14_1","doi-asserted-by":"publisher","DOI":"10.1177\/1756286419832242"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0161-5890(96)00078-8"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(17)30369-1"},{"key":"e_1_2_7_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26447"},{"key":"e_1_2_7_18_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.52.7.1487"},{"key":"e_1_2_7_19_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1749-6632.1998.tb11015.x"},{"key":"e_1_2_7_20_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.55.1.16"},{"key":"e_1_2_7_21_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1749-6632.2012.06817.x"},{"key":"e_1_2_7_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23988"},{"key":"e_1_2_7_23_1","doi-asserted-by":"publisher","DOI":"10.3390\/cells8070671"},{"issue":"15","key":"e_1_2_7_24_1","article-title":"Changes in concomitant immunosuppressive therapy use during a Phase 3 open\u2010label study of eculizumab in adults with generalized myasthenia gravis: an interim analysis","volume":"92","author":"Nowak RJ","year":"2019","journal-title":"Neurology"}],"container-title":["Annals of Clinical and Translational Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/acn3.51376","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/acn3.51376","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/acn3.51376","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,5,20]],"date-time":"2024-05-20T08:24:29Z","timestamp":1716193469000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/acn3.51376"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,5,27]]},"references-count":23,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2021,7]]}},"alternative-id":["10.1002\/acn3.51376"],"URL":"https:\/\/doi.org\/10.1002\/acn3.51376","archive":["Portico"],"relation":{},"ISSN":["2328-9503","2328-9503"],"issn-type":[{"value":"2328-9503","type":"print"},{"value":"2328-9503","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,5,27]]},"assertion":[{"value":"2020-11-17","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-04-14","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-05-27","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}