依库珠单抗治疗胸腺瘤相关重症肌无力：一项真实世界队列研究。
Eculizumab in thymoma-associated myasthenia gravis: a real-world cohort study.
作者信息
Jin Lei, He Dingxian, Zeng Quantao, Tan Song, Shi Jianquan, Liu Ying, Zou Zhangyu, Song Jie, Yan Chong, Huan Xiao, Wang Yuan, Yang Lei, Xi Jianying, Wu Zongtai, Liu Ziqi, Zheng Jianming, Zhao Chongbo, Chu Xianglin, Luo Sushan
机构信息
Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.
Department of Neurology, Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, China.
出版信息
Ther Adv Neurol Disord. 2024 Dec 25;17:17562864241309431. doi: 10.1177/17562864241309431. eCollection 2024.
BACKGROUND
Thymoma-associated myasthenia gravis (TAMG) is a subtype of myasthenia gravis (MG) that is associated with more severe symptoms and a relatively poor prognosis. Eculizumab, an inhibitor to target human C5 component of the complement cascade, is considered a treatment option for refractory generalized MG (gMG).
OBJECTIVES
To explore the safety and efficacy of eculizumab in patients with TAMG.
DESIGN
This is an observational multicenter real-world cohort study to assess TAMG who were treated with eculizumab from June 2023 to June 2024.
DATA SOURCES AND METHODS
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.
RESULTS
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 ± 12.1 years and an average disease duration of 4.0 ± 4.3 years. 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 ± 4.7 (baseline: 11.7 ± 6.0), and the corticosteroid dose reduced to 23.2 ± 26.5 mg (baseline: 41.8 ± 63.9 mg). 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.
CONCLUSION
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.
TRIAL REGISTRATION
NCT04535843.
背景
胸腺瘤相关重症肌无力（TAMG）是重症肌无力（MG）的一种亚型，其症状更为严重，预后相对较差。依库珠单抗是一种靶向补体级联反应人类C5成分的抑制剂，被认为是难治性全身型MG（gMG）的一种治疗选择。
目的
探讨依库珠单抗治疗TAMG患者的安全性和有效性。
设计
这是一项观察性多中心真实世界队列研究，旨在评估2023年6月至2024年6月接受依库珠单抗治疗的TAMG患者。
数据来源与方法
前瞻性收集与胸腺瘤相关多器官自身免疫（TAMA）相关的临床特征、重症肌无力日常生活活动（MG-ADL）评分以及治疗中出现的不良事件（TEAE）发生率。
结果
总体而言，5个研究中心的42例gMG患者接受了依库珠单抗治疗，最终纳入22例TAMG患者。该队列的平均年龄为51.5±12.1岁，平均病程为4.0±4.3年。关于胸腺瘤，世界卫生组织（WHO）组织学分类主要为B2和B3（63.7%），马萨oka分期主要为IV期（45.5%）。9名参与者（40.9%）从艾加莫德转换为依库珠单抗，旨在获得更好的临床改善并减少类固醇使用。到第12周时，MG-ADL评分降至4.8±4.7（基线：11.7±6.0），皮质类固醇剂量降至23.2±26.5mg（基线：41.8±63.9mg）。2例TAMA患者的皮肤病变和血小板减少症有显著改善。记录了2例TEAE，包括新型冠状病毒肺炎和唇疱疹感染。4例患者（18.2%）因胸腺瘤转移死于呼吸或循环衰竭。
结论
这项真实世界研究证明了依库珠单抗在控制TAMG症状和减少皮质类固醇使用方面的有效性。它也可能是难治性TAMG和TAMA的一种治疗选择。
试验注册号
NCT04535843。
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