Evidence map›Paper›PMID 39812736›Full record

ArticleNeurology and therapy2025

Content Validity of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia.

Michele Potashman, Katja Rudell, Naomi Suminski, Rinchen Doma, Maggie Heinrich, Linda Abetz-Webb, Melissa Wolfe Beiner, Vlad Coric, Liana S Rosenthal, Sheng-Han Kuo and 9 more

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In one paragraph

Article in Neurology and therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. 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

19 authors.

Michele PotashmanBiohaven Pharmaceuticals, Inc., 215 Church Street, New Haven, CT, 06510, USA. michele.potashman@biohavenpharma.com.ORCID http://orcid.org/0000-0001-7389-3354
Katja RudellCOA Science, Parexel International, London, UK.
Naomi SuminskiCOA Science, Parexel International, London, UK.
Rinchen DomaCOA Science, Parexel International, London, UK.
Maggie HeinrichCOA Science, Parexel International, London, UK.
Linda Abetz-WebbPatient-Centered Outcomes Assessments, Macclesfield, UK.
Melissa Wolfe BeinerBiohaven Pharmaceuticals, Inc., 215 Church Street, New Haven, CT, 06510, USA.
Vlad CoricBiohaven Pharmaceuticals, Inc., 215 Church Street, New Haven, CT, 06510, USA.
Liana S RosenthalDepartment of Neurology, Johns Hopkins School of Medicine, Baltimore, MD, USA.
Sheng-Han KuoDepartment of Neurology, Columbia University, New York, NY, USA.
Theresa ZesiewiczDepartment of Neurology and USF Ataxia Research Center, University of South Florida, Tampa, FL, USA.
Terry D FifeBarrow Neurological Institute, Phoenix, AZ, USA.
Bart van de WarrenburgDepartment of Neurology, Radboud University Medical Center, Nijmegen, Netherlands.
Giovanni RistoriDepartment of Neurosciences, Mental Health and Sensory Organs, Sant'Andrea Hospital, Sapienza University of Rome, Rome, Italy.
Matthis SynofzikDivision of Translational Genomics of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research, University of Tübingen, Tübingen, Germany.
Sub SubramonyDepartment of Neurology, University of Florida College of Medicine, Gainesville, FL, USA.
Susan PerlmanDepartment of Neurology, UCLA School of Medicine, Los Angeles, CA, USA.
Jeremy D SchmahmannLaboratory for Neuroanatomy and Cerebellar Neurobiology, Department of Neurology, Ataxia Center, Massachusetts General Hospital, Boston, MA, USA.
Gil L'ItalienBiohaven Pharmaceuticals, Inc., 215 Church Street, New Haven, CT, 06510, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionThe Friedreich Ataxia Rating Scale-Activities of Daily Living (FARS-ADL) is a validated and highly utilized measure for evaluating patients with Friedreich Ataxia. While construct validity of FARS-ADL has been shown for spinocerebellar ataxia (SCA), content validity has not been established.

methodsIndividuals with SCA1 or SCA3 (n = 7) and healthcare professionals (HCPs) with SCA expertise (n = 8) participated in qualitative interviews evaluating the relevance, clarity, and clinical meaningfulness of FARS-ADL for assessment of individuals with SCA. Interviews were recorded, transcribed, coded, and analyzed by ATLAS.Ti v22 software.

resultsFARS-ADL concepts most frequently reported by individuals with SCA were difficulty walking (n = 7/7), falls (n = 6/7), speech difficulties (n = 4/7), and swallowing (n = 3/7). Individuals with SCA reported that all FARS-ADL items were relevant; Gait and Walking (n = 7/7), Bladder Function (n = 6/7), and Falling (n = 6/7) were considered extremely relevant. All HCPs (n = 8/8) reported that most FARS-ADL items were relevant to individuals with SCA; Quality of Sitting Position was considered least relevant. HCPs reported meaningful change as 1-2 point score change in individual FARS-ADL items (n = 7/7), 1-3 point change in total score (n = 6/6), and stability on any item and/or total score over ≥ 1 year, depending on SCA subtype (n = 5/8). Cognitive debriefing supported clarity and comprehension of FARS-ADL. Suggested improvements included refining response options for Dressing, Falling, Walking, and Bladder Function items.

conclusionThe findings confirm the content validity of most FARS-ADL items for use in individuals with mild-to-moderate SCA1 and SCA3, and offer suggested improvements for response options.

Indexed as

Content validityFARS-ADLFriedreich Ataxia Rating ScaleSpinocerebellar ataxia

Identifiers

PMID39812736
PMCPMC11906946

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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.