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However, the safety and efficacy profiles from prospective studies are still lacking. <\/jats:p><jats:p> This study aimed to explore eculizumab\u2019s safety and efficacy in a prospective case series of patients with refractory MC. We followed a series of anti-acetylcholine receptor (AChR) antibody-positive myasthenia gravis (MG) patients who received eculizumab as an add-on therapy for 12\u2009weeks during MC to facilitate the weaning process and reduced disease activity. Serum anti-AChR antibodies and peripheral immune molecules associated with the complement pathway were evaluated before and after eculizumab administration. Compared to the baseline Myasthenia Gravis Foundation of America (MGFA)-quantitative MG test (QMG) scores (22.25\u2009\u00b1\u20094.92) and MG-activities of daily living (MG-ADL; 18.25\u2009\u00b1\u20092.5) scores at crisis, improvements were observed from 4\u2009weeks (14.5\u2009\u00b1\u200910.47 and 7.5\u2009\u00b1\u20097.59, respectively) through 12\u2009weeks (7.5\u2009\u00b1\u20095.74 and 2.25\u2009\u00b1\u20093.86, respectively) post-treatment. Muscle strength consistently improved across ocular, bulbar, respiratory, and limb\/gross domain groups. One patient died of cardiac failure at 16\u2009weeks. Three cases remained in remission at 24\u2009weeks, with a mean QMG score of 2.67\u2009\u00b1\u20092.89 and ADL score of 0.33\u2009\u00b1\u20090.58. No significant side effects were reported. Serum CH50 and soluble C5b-9 levels significantly declined, while there were no significant changes in serum anti-AChR antibody levels, C1q, C5a levels, or peripheral lymphocyte proportions. Eculizumab was well tolerated and showed efficacy in this case series. Large prospective cohort studies with extended follow-up periods are needed to further explore the safety and efficacy profile in real-world practice. <\/jats:p>","DOI":"10.1177\/17562864241261602","type":"journal-article","created":{"date-parts":[[2024,7,26]],"date-time":"2024-07-26T14:00:43Z","timestamp":1722002443000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":22,"title":["The safety and efficacy profile of eculizumab in myasthenic crisis: a prospective small case series"],"prefix":"10.1177","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0009-0003-5298-3993","authenticated-orcid":false,"given":"Jie","family":"Song","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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