Wang Shuai, Zhu Min, Dong Jihong, Zhang Yong, Luo Sushan, Jiang Jiahao, Cheng Zhaozhao, Li Zhijun, Yang Wenping, Yu Yue, Liu Zhengcheng, Fan Jiang, Xu Xiangnan, Liu Peipei, Zhang Zhouao, Can Fang, Liang Fei, Jiang Xifei, Tan Lijie, Ding Jianyong
Department of Thoracic Surgery, Zhongshan Hospital, Fudan University, Shanghai, People's Republic of China.
Department of Thoracic Surgery, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, People's Republic of China.
J Thorac Oncol. 2025 Aug;20(8):1120-1130. doi: 10.1016/j.jtho.2025.04.014. Epub 2025 May 2.
This study reports the primary perioperative outcomes of efgartigimod in patients with thymoma-associated myasthenia gravis (TAMG).
We conducted a prospective, single-arm clinical trial to evaluate the perioperative use of efgartigimod in acetylcholine receptor antibody-positive (AChR-Ab+) generalized TAMG. Efgartigimod was administered intravenously at a dose of 10 mg/kg on days 1, 8, 15, and 22, totaling four doses, with thymomectomy performed on day 9.
A total of 40 patients with TAMG were enrolled, with a median age of 46 years. The predominant thymomectomy approach was T2b minimally invasive thymectomy, performed in 21 of 40 patients (52.5%). All patients were classified as responders on the Myasthenia Gravis Activities of Daily Living, Quantitative Myasthenia Gravis, and Myasthenia Gravis Composite scale, demonstrating improvement across ocular, bulbar, limb, and respiratory muscle strength. At visit 4, myasthenic symptoms were obviously relieved, with reductions of 4.6 on Myasthenia Gravis Activities of Daily Living compared with baseline. Significant reductions were noted in both serum immunoglobulin G and AChR-Ab levels, without affecting other immunoglobulin classes or complement levels, after efgartigimod administration. The mean total postoperative hospitalization stay was 7.1 days. The total incidence of all-grade treatment-emergent adverse events was 62.5%, and the most treatment-emergent adverse events were reported to be mild. Postoperative myasthenic crisis occurred in four patients (10.0%) who underwent extended thymectomy.
The satisfactory outcomes and rapid neurologic remission demonstrated efgartigimod had favorable safety and efficacy profiles during the perioperative period of thymomectomy for TAMG. These findings support the potential of efgartigimod as a novel perioperative treatment option for TAMG.
NCT06221501.
本研究报告了艾加莫德治疗胸腺瘤相关性重症肌无力（TAMG）患者的围手术期主要结局。
我们开展了一项前瞻性单臂临床试验，以评估艾加莫德在乙酰胆碱受体抗体阳性（AChR-Ab+）的全身性TAMG患者围手术期的应用情况。艾加莫德于第1、8、15和22天静脉给药，剂量为10 mg/kg，共四剂，并于第9天进行胸腺切除术。
共纳入40例TAMG患者，中位年龄46岁。主要的胸腺切除方法是T2b微创胸腺切除术，40例患者中有21例（52.5%）采用此方法。所有患者在重症肌无力日常生活活动、重症肌无力定量和重症肌无力综合量表上均被分类为反应者，表明眼肌、延髓肌、肢体肌和呼吸肌力量均有改善。在第4次访视时，重症肌无力症状明显缓解，与基线相比，重症肌无力日常生活活动评分降低了4.6分。给予艾加莫德后，血清免疫球蛋白G和AChR-Ab水平均显著降低，且不影响其他免疫球蛋白类别或补体水平。术后平均总住院天数为7.1天。所有级别的治疗中出现的不良事件总发生率为62.5%，且报告的大多数治疗中出现的不良事件为轻度。4例（10.0%）接受扩大胸腺切除术的患者发生了术后重症肌无力危象。
令人满意的结局和快速的神经功能缓解表明，艾加莫德在TAMG胸腺切除术围手术期具有良好的安全性和疗效。这些发现支持了艾加莫德作为TAMG新型围手术期治疗选择的潜力。
NCT06221501。
