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Updated: Nov 15, 2025

Induction and Clinical Scoring of Chronic-Relapsing Experimental Autoimmune Encephalomyelitis
Published on: July 4, 2007
Tofacitinib treatment for refractory autoimmune encephalitis.
Yoonhyuk Jang1, Woo-Jin Lee1, Han Sang Lee1
1Department of Neurology, Seoul National University Hospital, Seoul, South Korea.
Tofacitinib, a Janus kinase (JAK) inhibitor, shows promise for treating difficult autoimmune encephalitis cases. This immunotherapy effectively crosses the blood-brain barrier (BBB), offering a new option for patients unresponsive to standard treatments.
Area of Science:
- Neuroimmunology
- Pharmacology
Background:
- Intractable autoimmune encephalitis requires novel therapies that penetrate the blood-brain barrier (BBB).
- Tofacitinib, a Janus kinase (JAK) inhibitor, crosses the BBB and is used for refractory immune-mediated diseases.
Observation:
- A retrospective study analyzed eight patients with refractory autoimmune encephalitis treated with tofacitinib (5 mg twice daily).
- Patients received tofacitinib after insufficient response to conventional multimodal immunotherapies.
Findings:
- Two patients (25%) showed good response (CGI-I=1 or 2), three (37.5%) had partial response (CGI-I=3), and three (37.5%) showed no improvement (CGI-I=4).
- Good responders experienced improvement in chronic autoimmune meningoencephalitis and cessation of refractory status epilepticus in anti-myelin oligodendrocyte glycoprotein (MOG)-associated disorder.
- No serious side effects were reported during tofacitinib treatment.
Implications:
- Tofacitinib demonstrates potential as a therapeutic option for central nervous system autoimmune diseases.
- Further research is warranted to explore tofacitinib's efficacy and safety in a larger cohort of patients with autoimmune encephalitis.
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