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During the open-label extension, the proportion of patients in the placebo\/eculizumab group who achieved \u2018minimal symptom expression\u2019 increased after initiating eculizumab treatment and was sustained through 130\u00a0weeks of open-label eculizumab (MG-ADL: 1.7\u00a0to\u00a027.8%; MG-QOL15: 1.7\u00a0to\u00a019.4%). At extension study week 130, similar proportions of patients in the eculizumab\/eculizumab and placebo\/eculizumab groups achieved \u2018minimal symptom expression\u2019 (MG-ADL: 22.9% and 27.8%, respectively, <jats:italic>p<\/jats:italic>\u2009=\u20090.7861; MG-QOL15: 14.3% and 19.4%, respectively, <jats:italic>p<\/jats:italic>\u2009=\u20090.7531). The long-term tolerability of eculizumab was consistent with previous reports.<\/jats:p>\n<\/jats:sec><jats:sec>\n<jats:title>Conclusions<\/jats:title>\n<jats:p>Patients with AChR+ refractory gMG who receive eculizumab can achieve sustained \u2018minimal symptom expression\u2019 based on patient-reported outcomes. \u2018Minimal symptom expression\u2019 may be a useful tool in measuring therapy effectiveness in gMG.<\/jats:p>\n<\/jats:sec><jats:sec>\n<jats:title>Trial registration<\/jats:title>\n<jats:p>ClinicalTrials.gov NCT01997229, NCT02301624.<\/jats:p>\n<\/jats:sec>","DOI":"10.1007\/s00415-020-09770-y","type":"journal-article","created":{"date-parts":[[2020,3,18]],"date-time":"2020-03-18T15:02:21Z","timestamp":1584543741000},"page":"1991-2001","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":94,"title":["\u2018Minimal symptom expression\u2019 in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab"],"prefix":"10.1007","volume":"267","author":[{"name":"The REGAIN study group","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6144-8544","authenticated-orcid":false,"given":"John","family":"Vissing","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0037-454X","authenticated-orcid":false,"given":"Saiju","family":"Jacob","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4320-7557","authenticated-orcid":false,"given":"Kenji P.","family":"Fujita","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5840-4089","authenticated-orcid":false,"given":"Fanny","family":"O\u2019Brien","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James F.","family":"Howard","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2020,3,18]]},"reference":[{"key":"9770_CR1","first-page":"255","volume":"86","author":"J Suh","year":"2013","unstructured":"Suh J, Goldstein JM, Nowak RJ (2013) Clinical characteristics of refractory myasthenia gravis patients. 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J.V. has received research and travel support, and\/or speaker honoraria from Alexion Pharmaceuticals and Sanofi\/Genzyme, and has served on advisory boards or as a consultant for Asklepios Biopharmaceuticals, Audentes Therapeutics, Novartis Pharma AG, PTC Therapeutics, Roche, Sanofi\/Genzyme, Santhera Pharmaceuticals, Sarepta Therapeutics, and Stealth Biotherapeutics within the past 3\u00a0years. S.J. is a member of an international advisory board for Alexion Pharmaceuticals, has been an advisory board member for Alnylam Pharmaceuticals and Argenx BVBA, has received speaker fees from Terumo BCT, and has received research support from the Wellcome Trust Clinical Research Facility and Centre for Rare Diseases at the University Hospitals Birmingham, UK. K.P.F. was employed by and owns stock in Alexion Pharmaceuticals and is employed by Alnylam Pharmaceuticals. F.O\u2019B. is employed by, and owns stock in, Alexion Pharmaceuticals. J.F.H. has received research support from Alexion Pharmaceuticals, argenx BVBA, the Centers for Disease Control and Prevention (Atlanta, GA, USA), the Muscular Dystrophy Association, the National Institutes of Health (including the National Institute of Neurological Disorders and Stroke and the National Institute of Arthritis and Musculoskeletal and Skin Diseases) and Ra Pharmaceuticals; has received honoraria from Alexion Pharmaceuticals; and has received non-financial support from Alexion Pharmaceuticals, argenx BVBA, Ra Pharmaceuticals and Toleranzia.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"This study was approved by the appropriate ethics committees and has been performed in accordance with the ethical standards laid down in the 1964 Declaration of Helsinki and its later amendments. All participants gave their informed consent prior to inclusion in the study.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}}]}}