Evidence map›Paper›PMID 41795178›Full record

ArticleMuscle & nerve2026

Experience Using Efgartigimod to Treat Juvenile Myasthenia Gravis in China: A Multicenter Retrospective Study.

Jing Lin, Guoyan Qi, Susan Luo, Qilong Jiang, Bitao Bu, Min Liu, Yuzhen Wei, Meigui Han, Junhong Yang, Zhilan Zhao and 5 more

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

Article in Muscle & nerve, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

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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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

15 authors.

Jing LinDepartment of Neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Guoyan QiDepartment of Neurology, Shijiazhuang City People's Hospital, Shijiazhuang, China.
Susan LuoHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.ORCID https://orcid.org/0000-0002-9033-7568
Qilong JiangDepartment of Neurology, The First Affiliated Hospital of Chinese Medicine, Guangzhou University of Chinese Medicine, Guangzhou, China.
Bitao BuDepartment of Neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Min LiuDepartment of Neurology, The Affiliated Hospital of Qingdao University, Qingdao, China.
Yuzhen WeiDepartment of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
Meigui HanDepartment of Pediatrics, The First Affiliated Hospital of Xinxiang Medical University, Xinxiang, China.
Junhong YangDepartment of Neurology, The First Affiliated Hospital of Henan University of Traditional Chinese Medicine, Zhengzhou, China.
Zhilan ZhaoDepartment of Neurology, The Second Affiliated Hospital of Guiyang Medical University, Guiyang, China.
Lili ZhangDepartment of Neurology, Daping Hospital, Army Medical University, Chongqing, China.
Yifan ZhangDepartment of Neurology, The Affiliated Hospital of Guizhou Medical University, Guiyang, China.
Haoyou XuDepartment of Neurology, Guangdong Hospital of Traditional Chinese Medicine, Guangzhou, China.ORCID https://orcid.org/0000-0003-1497-022X
Xingjie HaoDepartment of Epidemiology and Biostatistics, School of Public Health, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Zhijun LiDepartment of Neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.ORCID https://orcid.org/0000-0001-7198-8632

Funding

Health Commission of Hubei Province scientific research project WJ2021M119National Natural Science Foundation of China 82371411Natural Science Foundation of Hubei Province 2020CFB744
6 · The paper itself

Abstract

INTRODUCTION/

aimsCurrent therapeutic management of juvenile myasthenia gravis (JMG) predominantly relies on conventional immunosuppressive therapies and expert consensus extrapolated from adult data, creating a critical gap in high-quality, pediatric-specific clinical evidence. The aim of this study was to assess the efficacy and safety of efgartigimod in the pediatric population.

methodsThis multicenter retrospective study enrolled JMG patients (under 18 years), from 12 Chinese centers, who received at least one dose of efgartigimod. Efgartigimod was administered as weekly intravenous infusions. Study visits included W0 (baseline: pretreatment) and W1-W4 (weekly posttreatment follow-ups). Efficacy was assessed via MG-related Activities of Daily Living (MG-ADL) score, Quantitative Myasthenia Gravis (QMG) score, and the MG Composite (MGC) scale.

resultsSeventeen JMG patients (3 males, 14 females) with a median disease duration of 23 months were included. At W0, 88.2% of patients were classified as Myasthenia Gravis Foundation of America (MGFA) class II-V and were anti-acetylcholine receptor (anti-AchR) antibody positive. After efgartigimod treatment, clinically meaningful improvement (CMI) was observed in 70.6% (W1), 73% (W2), and 91.7% (W4); 66.7% achieved minimal symptom expression by W4. MG-ADL and QMG scores decreased by 30% and 30.1% (W1) and by 87.6% and 71.8% (W4) compared to W0. No treatment-related adverse events occurred. DISCUSSION: Our study suggests efgartigimod is an effective therapeutic option for JMG, with favorable efficacy and safety profiles. Further studies are necessary to validate the efficacy and safety of efgartigimod in the pediatric population.

Indexed as

Immunosuppressive AgentsMyasthenia GravisActivities of Daily LivingAdolescentChildChild, PreschoolChinaFemaleHumansMaleRetrospective StudiesTreatment OutcomeImmunosuppressive Agentsefficacyefgartigimodjuvenile myasthenia gravismonoclonal antibodiesneonate fc receptor blockerssafety

Identifiers

PMID41795178
PMCPMC13138349

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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.