Association between rituximab treatment and outcome in nonparaneoplastic autoimmune cerebellar ataxia: an observational cohort study
- 1Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Abstract
Background: Autoimmune cerebellar ataxia (ACA) can cause substantial disability. Evidence regarding immunotherapy for ACA, including rituximab (RTX), remains limited. This study evaluated the association of RTX treatment with outcomes in nonparaneoplastic ACA patients.
Methods: ACA patients from Peking Union Medical College Hospital (April 2013–July 2025) were included if they received immunotherapy and had documented RTX exposure status and modified Rankin Scale (mRS) scores at baseline and at last follow-up. The primary outcome was ΔmRS, defined as the zenith mRS score minus the last follow-up mRS score. Secondary outcomes included favorable outcome (last follow-up mRS score of ≤ 2) and relapse. Subgroup analyses were performed in patients with and without antineural antibodies, and patients with zenith mRS scores of ≥ 4.
Results: Among 144 included patients, 49 were categorized into the RTX group. Compared with the non-RTX group, the RTX group had higher zenith and last follow-up mRS scores, more often experienced relapse during the disease course, and were more likely to receive pulse corticosteroid and intravenous immunoglobulin treatment. RTX treatment was not independently associated with ΔmRS ( β = − 0.185, 95% confidence interval [CI]: −0.721 to 0.350; p = 0.495) or relapse risk (HR: 0.782, 95% CI: 0.267 to 2.294; p = 0.654). Among patients with antineural antibodies, RTX treatment was associated with a numerically greater ΔmRS, although this association did not reach statistical significance ( β = 0.658, 95% CI: − 0.050 to 1.365; p = 0.068). Relapse was consistently associated with poorer outcomes, whereas response to first-line immunotherapy was associated with better functional recovery.
Discussion: In this real-world ACA cohort, we did not observe a significant association between RTX treatment and improved prognosis or reduced relapse risk, although RTX may be associated with greater mRS improvement in patients with antineural antibodies. Functional outcome analyses should be interpreted cautiously because RTX-treated patients had important baseline and disease-course imbalances suggestive of clinically more complicated and relapsing disease.
Related
- Rituximab-induced hypogammaglobulinemia in autoimmune cytopenia and associated diseases.
- Immune Checkpoint Inhibitor Therapy Triggers Autoimmune Cerebellar Ataxia with Anti-P/Q-type VGCC Antibody and Anti-mGluR5 Antibodies: A Case Report
- Immunophenotyping of B- and T-cell alterations in patients with autoimmune premature ovarian insufficiency following rituximab treatment
- Persistent hypogammaglobulinaemia after rituximab in children with difficult-to-treat nephrotic syndrome: a multicentre cross-sectional observational study
- Reduced Circulating Regulatory T Cells in Autoimmune and Chronic Idiopathic Neutropenia of Childhood: An Observational Study of Clinical and Laboratory Associations
BibTeX
@article{liu2026association,
title = {{Association between rituximab treatment and outcome in nonparaneoplastic autoimmune cerebellar ataxia: an observational cohort study}},
author = {Mange Liu and Le Zhang and Siyuan Fan and Haitao Ren and Pinfei Ni and Yalin Ju and Lin Bai and Hongzhi Guan},
journal = {Frontiers in Immunology},
year = {2026},
month = oct,
doi = {10.3389/fimmu.2026.1931030},
url = {https://www.frontiersin.org/articles/10.3389/fimmu.2026.1931030/full},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Association between rituximab treatment and outcome in nonparaneoplastic autoimmune cerebellar ataxia: an observational cohort study
AU - Mange Liu
AU - Le Zhang
AU - Siyuan Fan
AU - Haitao Ren
AU - Pinfei Ni
AU - Yalin Ju
AU - Lin Bai
AU - Hongzhi Guan
PY - 2026
DA - 2026/10/08
JO - Frontiers in Immunology
DO - 10.3389/fimmu.2026.1931030
UR - https://www.frontiersin.org/articles/10.3389/fimmu.2026.1931030/full
AB - Background: Autoimmune cerebellar ataxia (ACA) can cause substantial disability. Evidence regarding immunotherapy for ACA, including rituximab (RTX), remains limited. This study evaluated the association of RTX treatment with outcomes in nonparaneoplastic ACA patients. Methods: ACA patients from Peking Union Medical College Hospital (April 2013–July 2025) were included if they received immunotherapy and had documented RTX exposure status and modified Rankin Scale (mRS) scores at baseline and at last follow-up. The primary outcome was ΔmRS, defined as the zenith mRS score minus the last follow-up mRS score. Secondary outcomes included favorable outcome (last follow-up mRS score of ≤ 2) and relapse. Subgroup analyses were performed in patients with and without antineural antibodies, and patients with zenith mRS scores of ≥ 4. Results: Among 144 included patients, 49 were categorized into the RTX group. Compared with the non-RTX group, the RTX group had higher zenith and last follow-up mRS scores, more often experienced relapse during the disease course, and were more likely to receive pulse corticosteroid and intravenous immunoglobulin treatment. RTX treatment was not independently associated with ΔmRS ( β = − 0.185, 95% confidence interval [CI]: −0.721 to 0.350; p = 0.495) or relapse risk (HR: 0.782, 95% CI: 0.267 to 2.294; p = 0.654). Among patients with antineural antibodies, RTX treatment was associated with a numerically greater ΔmRS, although this association did not reach statistical significance ( β = 0.658, 95% CI: − 0.050 to 1.365; p = 0.068). Relapse was consistently associated with poorer outcomes, whereas response to first-line immunotherapy was associated with better functional recovery. Discussion: In this real-world ACA cohort, we did not observe a significant association between RTX treatment and improved prognosis or reduced relapse risk, although RTX may be associated with greater mRS improvement in patients with antineural antibodies. Functional outcome analyses should be interpreted cautiously because RTX-treated patients had important baseline and disease-course imbalances suggestive of clinically more complicated and relapsing disease.
SN - 1664-3224
ER -