{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T23:30:59Z","timestamp":1793316659522,"version":"4.5.0"},"reference-count":30,"publisher":"Springer Science and Business Media LLC","issue":"5","license":[{"start":{"date-parts":[[2024,2,24]],"date-time":"2024-02-24T00:00:00Z","timestamp":1708732800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2024,2,24]],"date-time":"2024-02-24T00:00:00Z","timestamp":1708732800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100015661","name":"UCB Pharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100015661","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J Neurol"],"published-print":{"date-parts":[[2024,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec>\n                <jats:title>Background<\/jats:title>\n                <jats:p>Fatigue is a debilitating symptom of myasthenia gravis (MG). The impact of fatigue on MG can be assessed by Quality of Life in Neurological Disorders (Neuro-QoL) Short Form Fatigue scale. Transformation of raw Neuro-QoL fatigue scores to T-scores is a known approach for facilitating clinical interpretation of clinically meaningful and fatigue severity thresholds.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>In the Phase 3, double-blind, placebo-controlled RAISE study (NCT04115293), adults with acetylcholine receptor autoantibody-positive generalised MG (MG Foundation of America Disease Class II\u2013IV) were randomised 1:1 to daily subcutaneous zilucoplan 0.3\u00a0mg\/kg or placebo for 12\u00a0weeks. Patients completing RAISE could opt to receive zilucoplan 0.3\u00a0mg\/kg in an ongoing, open-label extension study, RAISE-XT (NCT04225871). In this post-hoc analysis, we evaluated the long-term effect of zilucoplan on fatigue in RAISE patients who entered RAISE-XT. We report change in Neuro-QoL Short Form Fatigue T-scores and fatigue severity levels from RAISE baseline to Week 60.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>Mean Neuro-QoL Short Form Fatigue T-scores improved from baseline to Week 12 in the zilucoplan group (<jats:italic>n<\/jats:italic>\u2009=\u200986) with a clinically meaningful difference versus placebo (<jats:italic>n<\/jats:italic>\u2009=\u200988; least squares mean difference:\u2009\u2212\u20093.61 (nominal <jats:italic>p<\/jats:italic>-value\u2009=\u20090.0060]), and these improvements continued further to Week 60. At Week 12, more patients on zilucoplan (<jats:italic>n<\/jats:italic>\u2009=\u200934, 47.2%) experienced improvements in\u2009\u2265\u20091 fatigue severity level from baseline versus placebo (<jats:italic>n<\/jats:italic>\u2009=\u200923, 28.4%; <jats:italic>p<\/jats:italic>\u2009=\u20090.017). At Week 60, most (<jats:italic>n<\/jats:italic>\u2009=\u200955, 65.5%) patients had mild fatigue or none.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusion<\/jats:title>\n                <jats:p>Treatment with zilucoplan demonstrated statistical and clinically meaningful improvements in fatigue scores and severity versus placebo during RAISE, which were sustained to Week 60 in RAISE-XT.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1007\/s00415-024-12209-3","type":"journal-article","created":{"date-parts":[[2024,2,24]],"date-time":"2024-02-24T06:02:30Z","timestamp":1708754550000},"page":"2758-2767","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["Improvement of fatigue in generalised myasthenia gravis with zilucoplan"],"prefix":"10.1007","volume":"271","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4064-1032","authenticated-orcid":false,"given":"Michael D.","family":"Weiss","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miriam","family":"Freimer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. 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Weiss has received honoraria for serving on scientific advisory boards for Alexion Pharmaceuticals, Immunovant, Ra Pharmaceuticals (now UCB Pharma), argenx, Biogen, Mitsubishi Tanabe Pharma and Amylyx; consulting honoraria from Cytokinetics and CSL Behring and speaker honoraria from Soleo Health. He also serves as a special government employee for the Food and Drug Administration. Miriam Freimer has served as a paid Consultant for argenx, UCB Pharma and Alexion Pharmaceuticals. She receives research support from the NIH, UCB Pharma, Janssen Pharmaceuticals, Alnylam, Avidity and Fulcrum. M. Isabel Leite is funded by the NHS (Myasthenia and Related Disorders Service and National Specialised Commissioning Group for Neuromyelitis Optica, UK) and by the University of Oxford, UK. She has been awarded research grants from the UK association for patients with myasthenia (Myaware) and the University of Oxford. She has received speaker honoraria or travel grants from Biogen, Novartis, UCB Pharma and the Guthy-Jackson Charitable Foundation. She serves on scientific or educational advisory boards for UCB Pharma, argenx and Viela Bio (now Horizon Therapeutics). Angelina Maniaol has received payment for travel, meeting attendance, consulting honoraria or advisory board participation from CSL Behring, Novartis, Biogen, argenx and UCB Pharma. Kimiaki Utsugisawa has served as a paid Consultant for UCB Pharma, argenx, Janssen Pharmaceuticals, Viela Bio (now Horizon Therapeutics), Chugai Pharmaceutical, HanAll Biopharma, Merck and Mitsubishi Tanabe Pharma. He has received speaker honoraria from argenx, Alexion Pharmaceuticals, UCB Pharma and the Japan Blood Products Organization. Jos Bloemers, Babak Boroojerdi and Natasa Savic are employees and shareholders of UCB Pharma. Emily Howard was an employee of Cogent working for UCB Pharma on a contract basis. James F. Howard Jr has received research support (paid to his institution) from Alexion AstraZeneca Rare Disease, argenx, Cartesian Therapeutics, the Centers for Disease Control and Prevention (Atlanta, GA, USA), the Myasthenia Gravis Foundation of America, 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), PCORI and UCB Pharma; honoraria from AcademicCME, Alexion AstraZeneca Rare Disease, argenx, Biologix Pharma, CheckRare CME, F. Hoffmann-La Roche, Horizon Therapeutics (now Amgen), MedScape CME, Merck EMD Serono, NMD Pharma, Novartis, PeerView CME, Physicians\u2019 Education Resource (PER) CME, PlatformQ CME, Regeneron Pharmaceuticals, Sanofi US, UCB Pharma and Zai Labs; and non-financial support from Alexion AstraZeneca Rare Disease, argenx, Cartesian Therapeutics, Toleranzia AB, UCB Pharma and Zai Labs.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"This study was conducted in accordance with ethical principles that have their origin in the Declaration of Helsinki and that are consistent with Good Clinical Practice. Institutional review boards or independent ethics committees provided written approval for the study protocol and all amendments. All patients provided written informed consent.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}}]}}