Efgartigimod Is an Effective Treatment for Triple-Seronegative Generalized Myasthenia Gravis: A Report of Three Patients.

IF 3.1 3区医学 Q2 CLINICAL NEUROLOGY

Muscle & Nerve Pub Date : 2025-08-01 Epub Date: 2025-05-20 DOI:10.1002/mus.28441

Jingyi Shen, Chi Ma, Ying Zhu, Ruixia Zhu

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Abstract

Introduction/aims: Efgartigimod has been approved for treating acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR-Ab+ gMG). The aim of this study was to report the therapeutic effect of efgartigimod in three patients with triple-seronegative (triple-SN) gMG.

Methods: The medical records of three patients with triple-SN gMG who received efgartigimod at a dose of 10 mg/kg weekly for four consecutive weeks (one treatment cycle) were retrospectively reviewed. The patients were evaluated using the quantitative MG (QMG) score and the MG activities of daily living (MG-ADL) scale.

Results: The first patient, who was refractory to conventional immunosuppressive therapies, demonstrated symptom improvement with a QMG reduction of 20 and an ADL reduction of 10 points following efgartigimod therapy. The second patient with a QMG score of 26 and an ADL score of 19, who showed a poor response to immunoglobulin therapy, achieved rapid symptom control after two cycles of efgartigimod treatment by week 10 (QMG: 2, ADL:1). The third patient, diagnosed with MG acute exacerbation (QMG: 6, ADL:5), responded favorably to efgartigimod therapy by week 4 (QMG:0, ADL:0).

Discussion: These patient reports provide preliminary evidence supporting the effectiveness of efgartigimod as a potential alternative treatment for patients with triple-SN gMG who do not respond adequately to traditional therapies. However, multicenter randomized controlled trials are necessary to further evaluate the efficacy and safety of efgartigimod in this patient population.

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埃夫加替莫德是治疗三血清阴性全身性重症肌无力的有效药物——附3例报告。

Efgartigimod已被批准用于治疗乙酰胆碱受体抗体阳性的全身性重症肌无力（AChR-Ab+ gMG）。本研究的目的是报告艾加替莫德治疗3例三血清阴性（triple-SN） gMG患者的疗效。方法：回顾性分析3例3 - sn型gMG患者连续4周（1个治疗周期）给予艾夫加替莫德10mg /kg /周治疗的病历。采用定量MG （QMG）评分和MG日常生活活动（MG- adl）量表对患者进行评估。结果：第一名患者对常规免疫抑制治疗难治性，经艾加替莫治疗后，QMG降低20分，ADL降低10分，症状得到改善。第二例患者QMG评分为26分，ADL评分为19分，对免疫球蛋白治疗反应较差，在第10周（QMG: 2分，ADL:1分）治疗2个周期后，症状得到快速控制。第三例患者被诊断为MG急性加重（QMG: 6, ADL:5），在第4周时对依加替莫德治疗反应良好（QMG:0, ADL:0）。讨论：这些患者报告提供了初步证据，支持efgartigimod作为对传统治疗反应不充分的三重sn型gMG患者的潜在替代治疗的有效性。然而，需要多中心随机对照试验来进一步评估艾加替莫德在该患者群体中的有效性和安全性。

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

Muscle & Nerve 医学-临床神经学

CiteScore

6.40

自引率

5.90%

发文量

287

审稿时长

3-6 weeks

期刊介绍： Muscle & Nerve is an international and interdisciplinary publication of original contributions, in both health and disease, concerning studies of the muscle, the neuromuscular junction, the peripheral motor, sensory and autonomic neurons, and the central nervous system where the behavior of the peripheral nervous system is clarified. Appearing monthly, Muscle & Nerve publishes clinical studies and clinically relevant research reports in the fields of anatomy, biochemistry, cell biology, electrophysiology and electrodiagnosis, epidemiology, genetics, immunology, pathology, pharmacology, physiology, toxicology, and virology. The Journal welcomes articles and reports on basic clinical electrophysiology and electrodiagnosis. We expedite some papers dealing with timely topics to keep up with the fast-moving pace of science, based on the referees'' recommendation.