Immune checkpoints inhibitors in the treatment of progressive multifocal leukoencephalopathy: A systematic review and meta-analysis
- 1Department of Neurology, Vanderbilt University Medical Center (VUMC), 1211 Medical Center Drive, Nashville, TN 37232 USA
- 2Department of Internal Medicine, University of California San Francesco, Fresno, CA USA
Abstract
Background: Progressive multifocal leukoencephalopathy (PML) is a rare but catastrophic demyelinating disease of the central nervous system (CNS) caused the JC virus (JCV). However, no effective treatments have been identified. Immune check point inhibitors (ICIs) are considered a potential treatment option. We conducted a systematic review and meta-analysis to pool the data from the literature that evaluates the efficacy of using ICI in the treatment of PML.
Methods: The search was conducted on January 24, 2026 using PubMed, Scopus, Cochrane Central Register of Controlled Trials (CENTRAL), and Embase. Studies were included if they included patients with PML and evaluated the efficacy of using ICIs including PD-1, PD-L1, and CTLA-4 inhibitors in PML treatment. The exposure of interest was the use of ICI in the treatment of PML. The outcomes of interest were survival, disability and cerebrospinal fluid (CSF) viral load. Rate and weighted mean difference (WMD) and their 95% confidence intervals (95%CI) were used as the effect size in the analysis.
Results: We included a total of 186 patients with PML from 52 articles. The survival rate among the included patients was 62% (95%CI: 52%-72%). There was no significant difference in disability between before and after the treatment with ICI (WMD =− 0.03; 95%CI: − 2.11–2.04). On the other hand, the treatment was associated with lower viral levels in the CSF (WMD = 69.20; 95%CI: 1.69–136.70).
Conclusions: Future randomized clinical trials or comparative observational studies that investigate the efficacy of using ICI in the treatment of PML compared to other treatment modalities are needed.
Supplementary Information: The online version contains supplementary material available at https://doi.org/10.1007/s10072-026-09498-z.
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BibTeX
@article{toubasi2026immune,
title = {{Immune checkpoints inhibitors in the treatment of progressive multifocal leukoencephalopathy: A systematic review and meta-analysis}},
author = {Ahmad Toubasi and Thuraya N. Al-Sayegh},
journal = {Neurological Sciences},
year = {2026},
month = nov,
doi = {10.1007/s10072-026-09498-z},
url = {https://doi.org/10.1007/s10072-026-09498-z},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Immune checkpoints inhibitors in the treatment of progressive multifocal leukoencephalopathy: A systematic review and meta-analysis
AU - Ahmad Toubasi
AU - Thuraya N. Al-Sayegh
PY - 2026
DA - 2026/11/04
JO - Neurological Sciences
DO - 10.1007/s10072-026-09498-z
UR - https://doi.org/10.1007/s10072-026-09498-z
AB - Background: Progressive multifocal leukoencephalopathy (PML) is a rare but catastrophic demyelinating disease of the central nervous system (CNS) caused the JC virus (JCV). However, no effective treatments have been identified. Immune check point inhibitors (ICIs) are considered a potential treatment option. We conducted a systematic review and meta-analysis to pool the data from the literature that evaluates the efficacy of using ICI in the treatment of PML. Methods: The search was conducted on January 24, 2026 using PubMed, Scopus, Cochrane Central Register of Controlled Trials (CENTRAL), and Embase. Studies were included if they included patients with PML and evaluated the efficacy of using ICIs including PD-1, PD-L1, and CTLA-4 inhibitors in PML treatment. The exposure of interest was the use of ICI in the treatment of PML. The outcomes of interest were survival, disability and cerebrospinal fluid (CSF) viral load. Rate and weighted mean difference (WMD) and their 95% confidence intervals (95%CI) were used as the effect size in the analysis. Results: We included a total of 186 patients with PML from 52 articles. The survival rate among the included patients was 62% (95%CI: 52%-72%). There was no significant difference in disability between before and after the treatment with ICI (WMD =− 0.03; 95%CI: − 2.11–2.04). On the other hand, the treatment was associated with lower viral levels in the CSF (WMD = 69.20; 95%CI: 1.69–136.70). Conclusions: Future randomized clinical trials or comparative observational studies that investigate the efficacy of using ICI in the treatment of PML compared to other treatment modalities are needed. Supplementary Information: The online version contains supplementary material available at https://doi.org/10.1007/s10072-026-09498-z.
SN - 1590-1874
SN - 1590-3478
ER -