ORIGINAL RESEARCH article
Front. Neurol.
Sec. Neuromuscular Disorders and Peripheral Neuropathies
Efgartigimod Non-Responders after the First Treatment Cycle in Generalized Myasthenia Gravis: A Retrospective Analysis of Predictive Factors
Provisionally accepted
Zhenyu Niu*
Haiqiang Jin*
Jianchun Wang
Ren Jingru
Feng Gao*
RAN LIUJing GuoNan Zhang
Yiming Zheng
Hongjun Hao- Department of Neurology, First Hospital, Peking University, Beijing, China
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Objective: This study aimed to identify predictors of suboptimal response to efgartigimod in patients with generalized myasthenia gravis (gMG). Methods: In this single-center retrospective study, 35 gMG patients treated with efgartigimod were categorized into responders (n=25) and non-responders (n=10). Responders were defined by a reduction of >2 points in MG-ADL or >3 points in QMG score after one cycle, whereas non-responders showed improvement below these thresholds and subsequently responded to eculizumab. Demographic, clinical, and serological features were compared using univariate and multivariate analyses. Results: Non-responders had higher baseline gross motor and respiratory sub-scores. Univariate analysis revealed that thymoma, non-thymoma tumors, thyroid disease, and other autoimmune diseases were more common in non-responders. Multivariate analysis indicated that combinations of these factors were associated with a high predicted probability of poor response (up to 97.9%). Interpretation: Comorbidities including thymoma, other tumors, thyroid disease, and additional autoimmune disorders may predict reduced response to efgartigimod in gMG patients. Systematic evaluation of these factors could help optimize treatment selection.
Keywords: Generalized Myasthenia Gravis, efgartigimod, treatment response, Eculizumab, predictors, Retrospective study
Received: 29 Sep 2025; Accepted: 27 Oct 2025.
Copyright: © 2025 Niu, Jin, Wang, Jingru, Gao, LIU, Guo, Zhang, Zheng and Hao. 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:
Zhenyu Niu, asterisk@pku.edu.cn
Haiqiang Jin, jhq911@sohu.com
Feng Gao, gaofh2011@126.com
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