Evidence map›Paper›PMID 40921886›Full record

Observational studyJournal of neurology2025

Factors associated with early response to efgartigimod in patients with generalized myasthenia gravis: a multicenter retrospective observational study.

Wenjia Zhu, Yuping Chen, Cong Tian, Jingsi Wang, Xinmei Wen, Hua Lou, Yuting Jiang, Li Di, Shanshan Gu, Yan Wang and 8 more

Abstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Journal of neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Trial
  2. Trial
  3. Article
  4. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

18 authors.

Wenjia ZhuDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Yuping ChenSenior Department of Neurology, The First Medical Center of PLA General Hospital, Beijing, China.
Cong TianResearch Institute of Neuromuscular and Neurodegenerative Disease, Department of Neurology, Qilu Hospital, Cheeloo College of Medicine, Shandong University, Jinan, China.
Jingsi WangDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Xinmei WenDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Hua LouDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Yuting JiangDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Li DiDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Shanshan GuCenter of Treatment of Myasthenia Gravis, People's Hospital of Shijiazhuang Affiliated to Hebei Medical, Shijiazhuang, China.
Yan WangSenior Department of Neurology, The First Medical Center of PLA General Hospital, Beijing, China.
Yaye WangDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Yan LuDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Min WangDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Min XuDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Hai ChenDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China.
Yuying ZhaoResearch Institute of Neuromuscular and Neurodegenerative Disease, Department of Neurology, Qilu Hospital, Cheeloo College of Medicine, Shandong University, Jinan, China.
Guoyan QiCenter of Treatment of Myasthenia Gravis, People's Hospital of Shijiazhuang Affiliated to Hebei Medical, Shijiazhuang, China. zzjwlsys@163.com.
Yuwei DaDepartment of Neurology, Xuanwu Hospital, National Center for Neurological Disorders, Capital Medical University, Beijing, China. dayuwei100@hotmail.com.ORCID http://orcid.org/0000-0002-0318-148X

Funding

National Key Research and Development Program of China 2017YFC0907700National Natural Science Foundation of China 62171299
6 · The paper itself

Abstract

background and purposeEfgartigimod has demonstrated efficacy in generalized myasthenia gravis (gMG) in both clinical trials and real-world studies. However, factors influencing early response have been less reported. This study aimed to evaluate the efficacy of efgartigimod in a multicenter gMG cohort and to identify the clinical factors associated with early therapeutic response.

methodsThis multicenter, real-world, retrospective, observational study included 115 gMG patients administered efgartigimod across four myasthenia gravis (MG) centers. Responders were defined as patients with a ≥ 2-point Myasthenia Gravis Activities of Daily Living (MG-ADL) or ≥ 3-point Quantitative Myasthenia Gravis Score (QMG) score reduction, while early responders were those achieving score reductions after the first infusion. Subgroup analyses were conducted based on immunosuppressant (IST) use. Logistic regression analysis was performed to identify factors associated with response to first efgartigimod infusion according to MG-ADL or QMG scores reduction. Variables were compared between responders and non-responders to identify early response factors.

resultsAfter the first infusion, 72.5% of patients achieved improvement in MG-ADL and 60.5% in QMG, with these rates increasing to 93.3% and 87.5% respectively by the fourth infusion. Efgartigimod demonstrated the most significant improvement in bulbar, limb, and ocular symptoms; however, there was no statistically significant improvement in respiratory symptoms occurred during the initial 4-week treatment period. Multivariate logistic analysis showed that short disease duration and high MG-ADL bulbar score at baseline indicated early response. High QMG bulbar score at baseline also indicated early response. Efficacy was independent of IST use. No patients discontinued treatment due to severe adverse events; minor side effects were not recorded.

conclusionsEfgartigimod demonstrated robust efficacy in gMG patients. Early response was linked to shorter disease duration and severe bulbar symptoms, which promotes the identification of patients who are likely to benefit quickly from efgartigimod.

Indexed as

Myasthenia GravisOutcome Assessment, Health CareActivities of Daily LivingAdultAgedFemaleHumansImmunosuppressive AgentsMaleMiddle AgedRetrospective StudiesTreatment OutcomeImmunosuppressive AgentsEfgartigimodGeneralized myasthenia gravisPredictorsRetrospective studyTreatment response

Identifiers

PMID40921886
PMCPMC12417274

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.