Evidence map›Paper›PMID 39876568›Full record

ArticleMovement disorders clinical practice2025

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, Rosalind S Chuang, Jeremy D Schmahmann, Susan L Perlman, Liana S Rosenthal, Delaram Safarpour, Hannah Casey, Fay B Horak, Christopher M Gomez

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Article in Movement disorders clinical practice, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

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2citing papers in PubMed
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1 · What the graph read from it

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2 · The registry

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3 · Its place in the literature

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2 citing papers in PubMed.

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4 · The record

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5 · Who and what money

Authors and funding

9 authors.

Andreea M Rawlings *Biogen, Cambridge, Massachusetts, USA.
Rosalind S Chuang *Biogen, Cambridge, Massachusetts, USA.
Jeremy D SchmahmannAtaxia Center, Division of Behavioral Neurology and Integrated Brain Health, Laboratory for Neuroanatomy and Cerebellar Neurobiology, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0003-0706-5125
Susan L PerlmanDepartment of Neurology, University of California, Los Angeles, California, USA.
Liana S RosenthalDepartment of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.ORCID https://orcid.org/0000-0001-5140-3967
Delaram SafarpourDepartment of Neurology, Oregon Health & Science University, Portland, Oregon, USA.ORCID https://orcid.org/0000-0003-2625-2049
Hannah CaseyDepartment of Neurology, The University of Chicago, Chicago, Illinois, USA.
Fay B HorakDepartment of Neurology, Oregon Health & Science University, Portland, Oregon, USA.
Christopher M GomezDepartment of Neurology, The University of Chicago, Chicago, Illinois, USA.

Funding

BiogenPfizer
6 · The paper itself

Abstract

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

objectivesTo 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.

methodsWe 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.

resultsAmong 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.

conclusionsPatient 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.

Indexed as

Patient Reported Outcome MeasuresSpinocerebellar AtaxiasAdultAgedFemaleHumansLongitudinal StudiesMaleMiddle AgedSeverity of Illness Indexataxiaclinical trial endpointslongitudinal studypatient‐reported outcomesstudy endpoints

Identifiers

PMID39876568
PMCPMC12070164

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