Wang Kan, Li Qiuju, Wu Yanan, Zhang Mengze, Wang Xiaokun, Peng Jing, Xie Chong, Xue Chunran, Gao Song, Gao Li, Yang Yiwei, Wang Yuhui, Zhang Lu, Hao Yong, Guan Yangtai
Department of Neurology, Renji Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China.
Department of Neurology, Punan Branch of Renji Hospital, Shanghai Jiaotong University School of Medicine (Punan Hospital in Pudong New District, Shanghai), Shanghai, China.
BMJ Neurol Open. 2025 Aug 3;7(2):e001180. doi: 10.1136/bmjno-2025-001180. eCollection 2025.
Myasthenia gravis (MG), an IgG-mediated autoimmune disorder targeting neuromuscular junctions, shows refractory in 12-20% of generalised MG (gMG) patients despite immunotherapies. Plasma exchange (PLEX) transiently depletes pathogenic mediators, while neonatal Fc receptor antagonists (eg, efgartigimod) offer novel therapeutic potential. Both PLEX and efgartigimod require adjunctive non-steroidal immunosuppressive therapy (NSIST) for sustained remission. This study aims to evaluate the effectiveness and safety of efgartigimod working as a bridge treatment after PLEX but before NSIST taking effect, while concurrently conducting a comparative analysis of clinical outcomes between PLEX and efgartigimod in gMG.
This multicentre, open-label, three-arm trial (n=45 gMG patients) assigns cohorts to PLEX+efgartigimod, PLEX alone or efgartigimod alone. The intervention comprises PLEX and/or efgartigimod. Oral glucocorticoids and cholinesterase inhibitors are allowed during this study. NSIST starts the day after completing PLEX or the second dose of efgartigimod. Outcomes are assessed at weeks 4, 8, 12, 16, 20, 24, 36 and 48. Primary endpoint: proportion achieving minimal symptom expression (MSE) at week 48. Secondary endpoints: median time to first MSE, adverse events (AE) incidence/severity, exacerbation rates, neurological functional assessment scores, cholinesterase inhibitor/corticosteroid usage, serological evolution of immunological markers. All AEs are systematically documented and causality-assessed.
Ethical clearance for this investigation was granted by the Institutional Review Board of Punan Hospital in accordance with Declaration of Helsinki principles. All enrolled participants will provide written informed consent through standardised documentation processes prior to study enrolment. The results will be accessible in peer-reviewed publications.
ChiCTR2500104662.
重症肌无力（MG）是一种以神经肌肉接头为靶点的IgG介导的自身免疫性疾病，尽管采用了免疫疗法，但12% - 20%的全身型重症肌无力（gMG）患者仍表现为难治性。血浆置换（PLEX）可短暂清除致病介质，而新生儿Fc受体拮抗剂（如艾加莫德）具有新的治疗潜力。PLEX和艾加莫德都需要辅助非甾体免疫抑制疗法（NSIST）以实现持续缓解。本研究旨在评估艾加莫德在PLEX后、NSIST起效前作为桥接治疗的有效性和安全性，同时对gMG患者中PLEX和艾加莫德的临床结局进行对比分析。
这项多中心、开放标签、三臂试验（n = 45例gMG患者）将患者队列分为PLEX + 艾加莫德组、单独PLEX组或单独艾加莫德组。干预措施包括PLEX和/或艾加莫德。本研究期间允许使用口服糖皮质激素和胆碱酯酶抑制剂。NSIST在完成PLEX或第二次注射艾加莫德后的次日开始使用。在第4、8、12、16、20、24、36和48周评估结局。主要终点：第48周达到最小症状表现（MSE）的比例。次要终点：首次达到MSE的中位时间、不良事件（AE）发生率/严重程度、病情加重率、神经功能评估评分、胆碱酯酶抑制剂/皮质类固醇使用情况、免疫标志物的血清学演变。所有AE均进行系统记录并评估因果关系。
本研究已获得浦南医院机构审查委员会根据《赫尔辛基宣言》原则给予的伦理批准。所有纳入的参与者将在研究入组前通过标准化文件流程提供书面知情同意书。研究结果将在同行评审的出版物上发表。
ChiCTR2500104662
