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Eculizumab, an inhibitor to target human C5 component of the complement cascade, is considered a treatment option for refractory generalized MG (gMG). <\/jats:p><\/jats:sec><jats:sec><jats:title>Objectives:<\/jats:title><jats:p> To explore the safety and efficacy of eculizumab in patients with TAMG. <\/jats:p><\/jats:sec><jats:sec><jats:title>Design:<\/jats:title><jats:p> This is an observational multicenter real-world cohort study to assess TAMG who were treated with eculizumab from June 2023 to June 2024. <\/jats:p><\/jats:sec><jats:sec><jats:title>Data sources and methods:<\/jats:title><jats:p> Clinical features associated with thymoma-associated multi-organ autoimmunity (TAMA), Myasthenia Gravis Activities of Daily Living (MG-ADL) score, and the incidence of treatment-emergent adverse events (TEAEs) were prospectively collected. <\/jats:p><\/jats:sec><jats:sec><jats:title>Results:<\/jats:title><jats:p> Overall, 42 patients with gMG were treated with eculizumab at 5 research centers, of whom 22 patients with TAMG were finally included. This cohort had a mean age of 51.5\u2009\u00b1\u200912.1\u2009years and an average disease duration of 4.0\u2009\u00b1\u20094.3\u2009years. Regarding thymomas, the World Health Organization (WHO) histological classification was primarily B2 and B3 (63.7%), and Masaoka staging was predominantly IV (45.5%). Nine participants (40.9%) switched from efgartigimod to eculizumab aiming at a better clinical improvement and reducing steroid use. By week 12, the MG-ADL score decreased to 4.8\u2009\u00b1\u20094.7 (baseline: 11.7\u2009\u00b1\u20096.0), and the corticosteroid dose reduced to 23.2\u2009\u00b1\u200926.5\u2009mg (baseline: 41.8\u2009\u00b1\u200963.9\u2009mg). Two patients with TAMA showed significant improvement in skin lesions and thrombocytopenia. Two TEAEs were recorded including COVID-19 and herpes labialis infection. Four patients (18.2%) died of respiratory or circulatory failure owing to thymoma metastasis. <\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion:<\/jats:title><jats:p> This real-world study demonstrates the efficacy of eculizumab in achieving symptom control and corticosteroid reduction for TAMG. It may also be a therapeutic option for refractory TAMG and TAMA. <\/jats:p><\/jats:sec><jats:sec><jats:title>Trial registration:<\/jats:title><jats:p> NCT04535843. <\/jats:p><\/jats:sec>","DOI":"10.1177\/17562864241309431","type":"journal-article","created":{"date-parts":[[2024,12,25]],"date-time":"2024-12-25T11:54:47Z","timestamp":1735127687000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":20,"title":["Eculizumab in thymoma-associated myasthenia gravis: a real-world cohort study"],"prefix":"10.1177","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9052-4590","authenticated-orcid":false,"given":"Lei","family":"Jin","sequence":"first","affiliation":[{"name":"Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, 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