Evidence map›Paper›PMID 41547654›Full record

ArticleNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics2026

Targeting pathogenic Th17.1 cells via JAK-STAT3 pathway: A novel approach with tofacitinib for refractory myasthenia gravis.

Rui Zhao, Chong Yan, Huahua Zhong, Xiao Huan, Lei Jin, Dingxian He, Jianying Xi, Yarong Li, Baoguo Xiao, Feifei Luo and 3 more

Registry-linked trialAbstract readClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Article in Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04431895 (A Prospective,Single-center,Single-Arm,Single-Blind Pilot Study To Investigate The Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis), which is not on this 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

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.

NCT04431895 early_phase1completednot on this map

A Prospective,Single-center,Single-Arm,Single-Blind Pilot Study To Investigate The Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis

TypeinterventionalSponsorHuashan HospitalRan2020 to 2024Enrolled19ConditionsMyasthenia Gravis, GeneralizedArmsTofacitinib 5 MG
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

13 authors.

Rui ZhaoHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China.
Chong YanHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China.
Huahua ZhongHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China.
Xiao HuanHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China.
Lei JinHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China.
Dingxian HeHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China.
Jianying XiHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China.
Yarong LiHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China.
Baoguo XiaoHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China; Institute of Neurology, Huashan Hospital, Institute of Brain Science and State Key Laboratory of Medical Neurobiology, Fudan University, Shanghai 200040, China.
Feifei LuoDepartment of Digestive Disorders, Huashan Hospital, Fudan University, Shanghai 200040, China.
Chongbo ZhaoHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China. Electronic address: zhao_chongbo@fudan.edu.cn.
Jie SongHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China. Electronic address: songj15@fudan.edu.cn.
Sushan LuoHuashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, Shanghai 200040, China. Electronic address: luosushan@fudan.edu.cn.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

There remains a critical unmet need for effective, accessible, and well-tolerated therapies for myasthenia gravis (MG) who are refractory to current immunotherapies. The janus kinase and signal transducer and activator of transcription (JAK-STAT) signaling pathway plays a pivotal role in maintaining immune homeostasis by regulating cytokine-mediated responses. However, insights into the involvement of the JAK-STAT signaling in MG pathogenesis are still preliminary. In this study, we observed elevated levels of JAK2 in MG and further assessed the clinical efficacy of the pan-JAK inhibitor, tofacitinib, administered over 24 weeks in a cohort of 19 patients with refractory MG (NCT04431895). Tofacitinib significantly reduces the glucocorticoid dose and improves MG-relevant clinical scores and quality of life. The immunomodulatory effects of tofacitinib were mediated through downregulation of p-STAT3, IL-6, and IL-23, resulting in suppression of pathogenic Th17.1 cells in MG. Collectively, our results suggest that a novel approach to suppress pathogenic Th17.1 cells via the JAK-STAT3 signalling pathway with tofacitinib is effective and well-tolerated for treating patients with refractory MG.

Indexed as

Janus KinasesMyasthenia GravisPiperidinesProtein Kinase InhibitorsPyrimidinesPyrrolesSTAT3 Transcription FactorAdultFemaleHumansMaleMiddle AgedSignal TransductionTh17 CellsJanus KinasesPiperidinesProtein Kinase InhibitorsPyrimidinesPyrrolesSTAT3 protein, humanSTAT3 Transcription FactortofacitinibJAK-STAT signaling pathwayMyasthenia gravisTh17.1 cellsTofacitinib

Identifiers

PMID41547654
PMCPMC12976549

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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.