Abstract
Background
Mycophenolate mofetil (MMF) is frequently used in the treatment of neurological autoimmune disorders. However, its effect on the relapse risk in anti-leucine-rich glioma–inactivated protein 1 (anti-LGI1) encephalitis is not well studied.
Methods
In this prospective observational cohort study, anti-LGI1 encephalitis patients were grouped according to MMF treatment status (MMF and non-MMF groups). The primary outcome was relapse after disease onset.
Results
A total of 83 patients were included, with a median onset age of 60 years. Fifty-four patients were men (65.1%). The MMF group comprised 28 patients and the non-MMF group comprised 55. Median follow-up from symptom onset was 26 months. Relapse occurred in 43 patients (51.8%). Median modified Rankin scale (mRS) score at enrollment was significantly higher in the MMF group than the non-MMF group (3 vs. 2; p = 0.001). Median mRS score at last follow-up was comparable between groups (1 vs. zero; p = 0.184). Both MMF treatment (HR 0.463; 95% CI, 0.231–0.929; p = 0.030) and cognitive impairment at enrollment (HR 3.391; 95% CI, 1.041–11.044; p = 0.043) were independent predictors of relapse. Starting immunotherapy before development of cognitive impairment trended towards reducing relapse risk. Outcome at last follow-up was good (mRS score 0–2) in all patients except for one in the non-MMF group. Adverse events associated with MMF treatment were mild and transient.
Conclusion
Although the outcome of anti-LGI1 encephalitis patients is generally favorable, relapse is common, especially in those with cognitive impairment. MMF treatment is well-tolerated and can significantly reduce the risk of relapse.
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Data availability
The data that support the findings of this study are available on request from the corresponding author.
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Funding
This study is funded by the National High Level Hospital Clinical Research Funding (2022-PUMCH-B-120) and CAMS Innovation Fund for Medical Sciences (CIFMS #2021-I2M-1-003).
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LM and GH designed the study. LM, XX, and FS recorded the clinical data. LM, RH, and ZY carried out the data analysis. LM wrote the first draft of the manuscript. Important data analysis suggestions were made by XX, FS, and RH. Manuscript revisions were made by GH.
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This study was approved by the Ethics Committee of Peking Union Medical College Hospital (JS-891). All included patients or their deputies provided written informed consent. The study was performed in accordance to the tenets of the Declaration of Helsinki.
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Liu, ., Xu, X., Fan, S. et al. Mycophenolate mofetil reduces the risk of relapse in anti-leucine-rich glioma–inactivated protein 1 encephalitis: a prospective observational cohort study. Neurol Sci 45, 253–260 (2024). https://doi.org/10.1007/s10072-023-06968-6
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DOI: https://doi.org/10.1007/s10072-023-06968-6