Evidence map›Paper›PMID 40888983›Full record

ArticleCerebellum (London, England)2025

Content Validity of the Spinocerebellar Ataxia Composite Score as a Measure of Disease Progression in Patients with Spinocerebellar Ataxia.

Michele Potashman, Maggie Heinrich, Katja Rudell, Linda Abetz-Webb, Naomi Suminski, Rinchen Doma, Kavita Jarodia, Mahak Jain, Chris Buckley, Melissa Wolfe-Beiner and 5 more

Abstract read
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In one paragraph

Article in Cerebellum (London, England), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Michele PotashmanBiohaven Pharmaceuticals, Inc., 215 Church St, New Haven, CT, USA. michele.potashman@biohavenpharma.com.
Maggie HeinrichCOA Science, Parexel International, London, UK.
Katja RudellCOA Science, Parexel International, London, UK.
Linda Abetz-WebbPatient-Centered Outcomes Assessment, Macclesfield, UK.
Naomi SuminskiCOA Science, Parexel International, London, UK.
Rinchen DomaCOA Science, Parexel International, London, UK.
Kavita JarodiaCOA Science, Parexel International, London, UK.
Mahak JainCOA Science, Parexel International, London, UK.
Chris BuckleyCOA Science, Parexel International, London, UK.
Melissa Wolfe-BeinerBiohaven Pharmaceuticals, Inc., 215 Church St, New Haven, CT, USA.
Vlad CoricBiohaven Pharmaceuticals, Inc., 215 Church St, New Haven, CT, USA.
Susan PerlmanDepartment of Neurology, UCLA School of Medicine, Los Angeles, CA, USA.
Liana RosenthalDepartment of Neurology, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Jeremy SchmahmannDepartment of Neurology, Massachusetts General Hospital, Boston, MA, USA.
Gilbert L'ItalienBiohaven Pharmaceuticals, Inc., 215 Church St, New Haven, CT, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Spinocerebellar ataxia (SCA) composite score (SCACOMS) is a statistically-derived composite measure comprising weighted items that are sensitive to change during early-stage disease. SCACOMS items and weights include the functional Scale for the Assessment and Rating of Ataxia Gait (12%), Stance (17%), Sitting (8%), and Speech (10%) items, and the Clinician Global Impression of Change (CGI) (53%). The content validity of SCACOMS is yet to be established. Semi-structured qualitative interviews were conducted with individuals with SCA (N = 24) and healthcare professionals (HCPs) who treat SCA (N = 2) to evaluate the relevance and weighting of SCACOMS items for assessment of SCA progression. Interviews were audio recorded, transcribed, coded, and analyzed using ATLAS.Ti v23 software, following established methods. SCACOMS items measured all sign and symptom concepts reported by > 50.0% of individuals with SCA, including difficulties with speech (79.2%), balance (75.0%), and gait (66.7%). Of SCACOMS items, individuals with SCA ranked Gait as most important for assessing disease progression (45.8%) and CGI as least important (58.3%). When weighting SCACOMS items, individuals with SCA assigned the highest weight to Gait (mean [standard deviation], 32.8% [13.24]) and the lowest weight to Sitting (12.9% [7.98]). HCPs varied the item weights dependent on SCA severity. Agreement with the statistically-derived weighting of SCACOMS varied, with 41.6% of individuals with SCA indicating that CGI weighting was too high. Overall, all participants indicated that SCACOMS could detect meaningful changes and/or disease stabilization. This study supports the content validity of SCACOMS in SCA; however, SCACOMS item weights may warrant adjustment.

Indexed as

Disease ProgressionSeverity of Illness IndexSpinocerebellar AtaxiasAdultAgedFemaleHumansMaleMiddle AgedReproducibility of ResultsYoung AdultClinical outcomes assessmentContent validitySpinocerebellar ataxiaSpinocerebellar ataxia composite score

Identifiers

PMID40888983
PMCPMC12401751

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.