Perioperative Safety and Efficacy of Efgartigimod for Thymoma-Associated Myasthenia Gravis: A Prospective, Multicenter, Phase II Clinical Trial

IF 20.8 1区医学 Q1 ONCOLOGY

Journal of Thoracic Oncology Pub Date : 2025-08-01 DOI:10.1016/j.jtho.2025.04.014

Shuai Wang PhD , Min Zhu PhD , Jihong Dong PhD , Yong Zhang PhD , Sushan Luo PhD , Jiahao Jiang PhD , Zhaozhao Cheng PhD , Zhijun Li PhD , Wenping Yang PhD , Yue Yu PhD , Zhengcheng Liu PhD , Jiang Fan PhD , Xiangnan Xu MD , Peipei Liu PhD , Zhouao Zhang MD , Fang Can PhD , Fei Liang PhD , Xifei Jiang PhD , Lijie Tan PhD , Jianyong Ding PhD

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Abstract

Introduction

This study reports the primary perioperative outcomes of efgartigimod in patients with thymoma-associated myasthenia gravis (TAMG).

Methods

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.

Results

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.

Conclusion

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.

ClinicalTrials.gov Identifier

NCT06221501.

Abstract Image

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依加替莫德治疗胸腺瘤相关性重症肌无力的围手术期安全性和有效性：一项前瞻性、多中心、II期临床试验

本研究报道了依加替莫德治疗胸腺瘤相关性重症肌无力（TAMG）患者围手术期的主要预后。方法：我们进行了一项前瞻性单臂临床试验，以评估依加替吉莫在乙酰胆碱受体抗体阳性（AChR-Ab+）全身性TAMG围手术期的应用。在第1、8、15和22天静脉给药，剂量为10mg /kg，共4次，第9天进行胸腺瘤切除术。结果：40例tam患者入组，中位年龄为46岁。40例患者中有21例（52.5%）采用T2b微创胸腺切除术。所有患者在重症肌无力日常生活活动（MG-ADL）、定量重症肌无力（QMG）和重症肌无力复合（MGC）量表上均被分类为应答者，显示眼部、球部、肢体和呼吸肌力量均有改善。在第4次就诊时，肌无力症状明显缓解，与基线相比MG-ADL降低了4.6。给药后血清IgG和AChR-Ab水平显著降低，不影响其他免疫球蛋白类或补体水平。术后平均总住院时间为7.1天。所有级别治疗中出现的不良事件（teae）的总发生率为62.5%，大多数teae报告为轻度。术后4例（10.0%）患者行延长胸腺切除术后出现肌无力危象。结论：依加替莫在胸腺瘤切除术围手术期具有良好的安全性和有效性，治疗效果满意，神经功能迅速缓解。这些发现支持了艾加替莫德作为一种新的tamm围手术期治疗选择的潜力。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Thoracic Oncology 医学-呼吸系统

CiteScore

36.00

自引率

3.90%

发文量

1406

审稿时长

13 days

期刊介绍： Journal of Thoracic Oncology (JTO), the official journal of the International Association for the Study of Lung Cancer,is the primary educational and informational publication for topics relevant to the prevention, detection, diagnosis, and treatment of all thoracic malignancies.The readship includes epidemiologists, medical oncologists, radiation oncologists, thoracic surgeons, pulmonologists, radiologists, pathologists, nuclear medicine physicians, and research scientists with a special interest in thoracic oncology.