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ORIGINAL RESEARCH article
Front. Immunol.
Sec. Autoimmune and Autoinflammatory Disorders : Autoimmune Disorders
Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1579859
This article is part of the Research Topic Autoantibodies and autoimmune neuromuscular disorders View all 6 articles
Efgartigimod as a fast-acting treatment in generalized very-late-onset myasthenia gravis
Provisionally accepted
Zhouao Zhang 1
Mingjin Yang 1,2,3 Xinyan Guo 1,2 Tianyu Ma 1,2 Zhouyi Wang 1,2,3 Tiancheng Luo 1,2,3 Deyou Peng 1,2 Xue Du 1,2
Huang Xiao-Yu 1
Yong Zhang 1*- 1 The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu Province, China
- 2 First Clinical Medical College, Xuzhou Medical University, Xuzhou, Jiangsu Province, China
- 3 Central Laboratory, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, China
Objective: Efgartigimod (EFG), a neonatal Fc receptor antagonist that facilitates the degradation of pathogenic immunoglobulin G, is approved for the treatment of generalized myasthenia gravis (MG). This study aims to evaluate the efficacy and safety of EFG in patients with very-late-onset myasthenia gravis (VLOMG).Methods: This study enrolled 15 consecutive patients diagnosed with VLOMG who received EFG treatment. Baseline demographic and clinical characteristics, as well as dynamic changes in the MG-specific activities of daily living (MG-ADL) score and quantitative MG (QMG) score, were systematically recorded.Results: Patients were stratified into two groups: a worse group (n = 8) and a new-diagnosed group (n = 7), the latter of which included 5 patients who had received monotherapy with pyridostigmine (Py) prior to EFG. At week 5, the mean changes in MG-ADL scores were -4.9 ± 3.3 in the overall VLOMG cohort, -6.1 ± 3.1 in the new-diagnosed group, -6.6 ± 3.6 in the mono-Py subgroup, and -3.8 ± 3.2 in the worse group. The clinical meaningful improvement (CMI) rate was 86.7% (13/15) in the overall cohort, 75.0% (6/8) in the worse group, and 100.0% (7/7) in the new-diagnosed group. During a mean follow-up time of 39.2 ± 16.2 weeks, symptoms remained stable in responsive patients, with various treatment strategies implemented following the fast-acting treatment of EFG. No adverse drug reactions were reported in this cohort.This study demonstrates that EFG is an effective and safe treatment for patients with VLOMG. EFG exhibits potential as an early, fast-acting treatment and may confer sustained clinical benefits in this patient population.
Keywords: Very-late-onset Myasthenia Gravis, fast-acting treatment, efgartigimod, MG-ADL, clinical meaningful improvement
Received: 19 Feb 2025; Accepted: 31 Mar 2025.
Copyright: © 2025 Zhang, Yang, Guo, Ma, Wang, Luo, Peng, Du, Xiao-Yu and Zhang. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Yong Zhang, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu Province, China
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