Abstract Details

Title Predictors of Prognosis in Autoimmune/Paraneoplastic Cerebellar Ataxia: A Retrospective Cohort

Topic Autoimmune Neurology

Presentation(s) P8 - Poster Session 8 (11:45 AM-12:45 PM)

Poster/Presentation
Number 1-012

Objective This study aims to characterize autoantibody-positive autoimmune cerebellar ataxia (ACA), identify factors associated with poor functional status, and assess functional outcomes using different metrics.

Background ACA commonly causes significant morbidity. Prognostic predictors may better inform timely and appropriate immunotherapy. Traditional functional outcome measures such as the modified Rankin Scale (mRS) and the Clinical Assessment Scale in Autoimmune Encephalitis (CASE) may not adequately capture cerebellar symptom changes.

Design/Methods This retrospective cohort study included adults (> 18 years) treated for seropositive ACA at Massachusetts General Hospital or Brigham and Women’s Hospital (2004– 2025). Clinical characteristics, treatments, and outcomes were compared between paraneoplastic and non-paraneoplastic cohorts. The patient with an immune-related adverse event (irAE) after immune checkpoint inhibitor exposure was analyzed separately. Time to wheelchair dependence was estimated with the Kaplan–Meier method and compared using the log-rank test. Univariable Cox proportional hazards regression was used to identify predictors of wheelchair dependence. Functional outcomes were assessed using mRS, CASE, and Brief Ataxia Rating Scale (BARS).

Results 19 seropositive ACA cases (median age 66 years; 68.4% female) were included. 11 (57.9%) were paraneoplastic, 7 (36.8%) were non-paraneoplastic, and 1 (5.3%) was irAE. All paraneoplastic patients were PCA1 autoantibody–positive; non-paraneoplastic cases had heterogeneous autoantibodies. Paraneoplastic patients were more often female (90.9% vs. 28.6%, p = 0.01), had worse functional outcomes by BARS at last follow-up (20 vs. 7, p < 0.01), and higher mortality (63.6% vs. 14.3%, p = 0.046). Acute symptom onset (< 2 weeks), paraneoplastic association, and PCA1 positivity were associated with wheelchair dependence. BARS appeared to monitor ACA symptoms more precisely than mRS or CASE.

Conclusions Non-paraneoplastic cases showed diverse anti-neuronal antibody profiles. Acute onset, paraneoplastic association, and PCA1 positivity were key predictors of severe functional decline. A cerebellar symptom–specific outcome measure such as BARS should be used to monitor ACA.

Authors/Disclosures
Sophia G. Cerroni PRESENTER	Miss Cerroni has nothing to disclose.
Yoji Hoshina, MD (University of Utah Health)	Dr. Hoshina has nothing to disclose.
Trevor Glenn, MD (Mass General Brigham)	Dr. Glenn has nothing to disclose.
Nupur N. Goel, MD (Alcott Apartments)	Dr. Goel has nothing to disclose.
Bruna Leles Vieira de Souza, MD (Work)	Miss Leles Vieira de Souza has nothing to disclose.
Joao Vitor Mahler, MD	Dr. Mahler has received research support from The Sumaira Foundation.
Mattia Wruble, MD	The institution of Dr. Wruble has received research support from Alexion. The institution of Dr. Wruble has received research support from Roche.
Giovanna Manzano, MD	Dr. Manzano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Gilead Sciences. Dr. Manzano has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for InfuCare Rx.

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