Evidence map›Paper›PMID 39735403›Full record

ArticleTherapeutic advances in neurological disorders2024

Eculizumab in thymoma-associated myasthenia gravis: a real-world cohort study.

Lei Jin, Dingxian He, Quantao Zeng, Song Tan, Jianquan Shi, Ying Liu, Zhangyu Zou, Jie Song, Chong Yan, Xiao Huan and 9 more

Registry-linked trialAbstract read
In one paragraph

Article in Therapeutic advances in neurological disorders, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04535843 (Precision Diagnosis and Prospective Cohort Study for Myasthenia Gravis), which is not on this map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing 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.

NCT04535843 recruitingnot on this map

Precision Diagnosis and Prospective Cohort Study for Myasthenia Gravis: Multicenter Analysis in China

Typeobservational_patient_registrySponsorHuashan HospitalRan2024 to 2028Enrolled500ConditionsMyasthenia GravisArmsBiomarkers and omics data
3 · Its place in the literature

Who cites it

13 citing papers in PubMed.

  1. Myasthenia Gravis and Thymoma.Muscle & nerve · 2026
    Review
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  7. Review
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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

19 authors.

Lei JinHuashan 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/0000-0002-9052-4590
Dingxian HeHuashan 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/0009-0005-9955-7179
Quantao ZengDepartment of Neurology, Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, China.
Song TanDepartment of Neurology, Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, China.
Jianquan ShiDepartment of Neurology, Nanjing First Hospital, Nanjing Medical University, Nanjing, China.ORCID https://orcid.org/0000-0003-0536-1131
Ying LiuDepartment of Neurology, The Second Hospital of Dalian Medical University, Dalian, China.
Zhangyu ZouDepartment of Neurology, Fujian Medical University Union Hospital, Fuzhou, China.
Jie SongHuashan 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/0000-0002-4572-5721
Chong YanHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.
Xiao HuanHuashan 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/0000-0002-6122-4804
Yuan WangDepartment of Blood Transfusion, Huashan Hospital, Fudan University, Shanghai, China.
Lei YangDepartment of Neurosurgery and Neurocritical Care, Huashan Hospital Fudan University, Shanghai, China.
Jianying XiHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai, China.
Zongtai WuFaculty of Biology, University of Cambridge, Cambridge, UK.
Ziqi LiuDepartment of Dermatology, Huashan Hospital, Fudan University, Shanghai, China.
Jianming ZhengDepartment of Infectious Diseases, Huashan Hospital, National Medical Center for Infectious Diseases, Fudan University, Shanghai, China.
Chongbo ZhaoHuashan 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/0000-0001-9481-1418
Xianglin ChuDepartment of Thoracic Surgery, Huashan Hospital, Fudan University, Shanghai 200040, China.
Sushan LuoHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, No.12 Urumqi Middle Road, Jing 'an District, Shanghai 200040, China.ORCID https://orcid.org/0000-0002-9033-7568

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Thymoma-associated myasthenia gravis (TAMG) is a subtype of myasthenia gravis (MG) that is associated with more severe symptoms and a relatively poor prognosis. Eculizumab, an inhibitor to target human C5 component of the complement cascade, is considered a treatment option for refractory generalized MG (gMG). Objectives: To explore the safety and efficacy of eculizumab in patients with TAMG. Design: This is an observational multicenter real-world cohort study to assess TAMG who were treated with eculizumab from June 2023 to June 2024. Data sources and methods: Clinical features associated with thymoma-associated multi-organ autoimmunity (TAMA), Myasthenia Gravis Activities of Daily Living (MG-ADL) score, and the incidence of treatment-emergent adverse events (TEAEs) were prospectively collected. Results: Overall, 42 patients with gMG were treated with eculizumab at 5 research centers, of whom 22 patients with TAMG were finally included. This cohort had a mean age of 51.5 ± 12.1 years and an average disease duration of 4.0 ± 4.3 years. Regarding thymomas, the World Health Organization (WHO) histological classification was primarily B2 and B3 (63.7%), and Masaoka staging was predominantly IV (45.5%). Nine participants (40.9%) switched from efgartigimod to eculizumab aiming at a better clinical improvement and reducing steroid use. By week 12, the MG-ADL score decreased to 4.8 ± 4.7 (baseline: 11.7 ± 6.0), and the corticosteroid dose reduced to 23.2 ± 26.5 mg (baseline: 41.8 ± 63.9 mg). Two patients with TAMA showed significant improvement in skin lesions and thrombocytopenia. Two TEAEs were recorded including COVID-19 and herpes labialis infection. Four patients (18.2%) died of respiratory or circulatory failure owing to thymoma metastasis. Conclusion: This real-world study demonstrates the efficacy of eculizumab in achieving symptom control and corticosteroid reduction for TAMG. It may also be a therapeutic option for refractory TAMG and TAMA. Trial registration: NCT04535843.

Indexed as

corticosteroideculizumabrefractoryTAMATAMG

Identifiers

PMID39735403
PMCPMC11672488

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Registered trials

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.