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Prognostic and relapsing factors of primary autoimmune cerebellar ataxia: a prospective cohort study

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Abstract

Objective

The objective of this study was to investigate the factors influencing relapse and prognosis in patients with primary autoimmune cerebellar ataxia (PACA), an area previously not well understood.

Methods

This prospective cohort study included patients who satisfied the modified diagnostic criteria of PACA. A modified Rankin scale score ≤ 2 at the last follow-up was defined as a favorable prognosis. Cox and Logistic regression were utilized to identify relapsing and prognostic factors, respectively.

Results

A total of 68 patients were included and 35.3% were male. The median onset age was 42.9 years (IQR 22.1–54.0). Neuronal autoantibodies were detected in 33 (50.8%) patients. Of the 65 patients who received first-line immunotherapy, 55 (84.6%) were responsive and 10 (15.4%) were not. Responsiveness to first-line immunotherapy emerged as an independent factor for favorable prognosis (HR 16.762; 95% CI 2.877–97.655; p = 0.002), as did the absence of peripheral neuropathy/radiculopathy (HR 14.286; 95% CI 2.41–83.333; p = 0.003). Relapses occurred in 19 (27.9%) patients. Onset age ≤ 43 years (HR 5.245; 95% CI 1.499–18.35; p = 0.009), presence of peripheral neuropathy/radiculopathy (HR 4.280; 95% CI 1.622–11.298; p = 0.003) and elevated cerebrospinal fluid (CSF) protein concentration (HR 3.443; 95% CI 1.083–10.951; p = 0.036) were statistically significant relapsing factors.

Conclusion

This study identified younger onset age, presence of peripheral neuropathy/radiculopathy and elevated CSF protein concentration as relapsing factors, and absence of peripheral neuropathy/radiculopathy and responsiveness to first-line immunotherapy as independent factors for favorable prognosis in PACA patients. These findings may guide individualized treatment strategies and potentially improve patient outcomes.

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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 National High-Level Hospital Clinical Research Funding (2022-PUMCH-B-120).

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Authors and Affiliations

  1. Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China

    Mange Liu, Haitao Ren, Siyuan Fan, Lin Bai & Hongzhi Guan

  2. Department of Cardiology, State Key Laboratory of Complex Severe and Rare Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China

    Lei Wang

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  1. Mange Liu
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  2. Haitao Ren
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  4. Siyuan Fan
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  6. Hongzhi Guan
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Contributions

LM and GH designed the study. LM, WL and BL recorded the clinical data. LM, BL and FS carried out the data analysis. LM wrote the first draft of the manuscript. RH was responsible for neuronal antibody testing. Important data analysis suggestions were made by RH, FS and GH. Manuscript revisions were made by GH.

Corresponding author

Correspondence to Hongzhi Guan.

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The authors report no conflicts of interest.

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Liu, M., Ren, H., Wang, L. et al. Prognostic and relapsing factors of primary autoimmune cerebellar ataxia: a prospective cohort study. J Neurol 271, 1072–1079 (2024). https://doi.org/10.1007/s00415-023-12128-9

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  • DOI: https://doi.org/10.1007/s00415-023-12128-9

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