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. 2025 May;12(5):638-647.
doi: 10.1002/mdc3.14323. Epub 2025 Jan 28.

Longitudinal Changes in Patient- and Clinical-Reported Outcomes in Early Spinocerebellar Ataxia Types 1, 2, 3, and 6 from the IDEA Study

Andreea M Rawlings #  1 , Rosalind S Chuang #  1 , Jeremy D Schmahmann  2 , Susan L Perlman  3 , Liana S Rosenthal  4 , Delaram Safarpour  5 , Hannah Casey  6 , Fay B Horak  5   7 , Christopher M Gomez  6
Affiliations

Longitudinal Changes in Patient- and Clinical-Reported Outcomes in Early Spinocerebellar Ataxia Types 1, 2, 3, and 6 from the IDEA Study

Andreea M Rawlings et al. Mov Disord Clin Pract. 2025 May.

Abstract

Background: Clinical outcomes assessments (COAs) in spinocerebellar ataxia (SCA) need to be standardized, ataxia-specific, sensitive to change, clinically relevant, and meaningful to patients.

Objectives: To evaluate the longitudinal 1- and 2-year performances of different patient reported outcomes, including the Patient Reported Outcome Measure of Ataxia (PROM-Ataxia), and clinician reported outcomes, including FARS and SARA, in those with early manifest symptoms of SCA 1, 2, 3, and 6.

Methods: We studied 53 patients with early stage SCA1-3 and SCA6 from The Instrumented Data Exchange for Ataxia Study and 24 age-matched healthy controls. Participants were seen every 6 months for 2 years. Mixed models were used to estimate change over 12- and 24-months of follow-up. Changes on the FARS-FS and PGI-C were used as anchors to estimate meaningful changes.

Results: Among persons with SCA, mean age was 48.7 years and mean SARA score was 9.3. Few measures showed statistically significant changes at 12 months. At 24-months, the FARS-ADL, PROM-Ataxia total, PROM-Ataxia physical, and PROM-Ataxia ADL scores showed the strongest associations of change.

Conclusions: Patient reported or derived outcome measures, such as FARS-ADL and ADL sub domain of the PROM-Ataxia, can capture longitudinal change in patients' symptom experience over a 2-year period and its impact on daily activities, even in those with early disease. More work is needed to identify outcomes that reliably capture change earlier.

Keywords: ataxia; clinical trial endpoints; longitudinal study; patient‐reported outcomes; study endpoints.

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Conflict of interest statement

Ethical Compliance Statement: This study was approved by University of Chicago BSD IRB Committee (IRB18‐1850) and by institutional review boards at each study site. Participants provided written informed consent. The IDEA study was sponsored by Biogen and Pfizer. We confirm that we have read the Journal's position on issues involved in ethical publication and affirm that this work is consistent with those guidelines.

Funding Sources and Conflict of Interest: The IDEA study was sponsored by Biogen and Pfizer. AMR has received no specific funding for this work and has no conflicts of interest relevant to this work. RSC has received no specific funding for this work and has no conflicts of interest relevant to this work. JDS has received no specific funding for this work. The Patient Reported Outcome Measure of Ataxia (PROM‐Ataxia) was developed by JDS and is copyrighted by the Massachusetts General Hospital Corporation together with Dr. Schmahmann. SLP has received no specific funding for this work and has no conflicts of interest relevant to this work. LSR received research support to conduct this investigation from Pfizer and Biogen. DS has received no specific funding for this work and has no conflicts of interest relevant to this work. HC has received no specific funding for this work and has no conflicts of interest relevant to this work. FH was CoPI of the IDEA study. FH is a part‐time scientific advisor to APDM Precision Motion, Clario whose sensors were used in this study. This potential conflict of interest is managed by OHSU. CMG has received no specific funding for this work and has no conflicts of interest relevant to this work.

Financial Disclosures for the Previous 12 Months: AMR is an employee of Biogen. RSC is an employee of Biogen. JDS is the site PI for Biohaven Pharmaceuticals clinical trials NCT03701399 and NCT02960893, consults for Biohaven Pharmaceuticals, receives royalties from Oxford University Press, Elsevier, MacKeith Press, and Springer, and is the inventor of the Brief Ataxia Rating Scale, Cerebellar Cognitive Affective/Schmahmann Syndrome Scale, the Patient Reported Outcome Measure of Ataxia, and the Cerebellar Neuropsychiatry Rating Scale which are licensed to the General Hospital Corporation. SLP has no additional disclosures to report. LSR has no additional disclosures to report. S has no additional disclosures to report. HC has no additional disclosures to report. FH receives support from the National Institutes of Health and the Michael J Fox Foundation. CMG has no additional disclosures to report.

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