Content Validity of the Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) Instrument in Spinocerebellar Ataxia

  1. Potashman, Michele
  2. Rudell, Katja
  3. Pavisic, Ivanna
  4. Suminski, Naomi
  5. Doma, Rinchen
  6. Heinrich, Maggie
  7. Abetz-Webb, Linda
  8. Beiner, Melissa Wolfe
  9. Kuo, Sheng-Han
  10. Rosenthal, Liana S.
  11. Zesiwicz, Theresa
  12. Fife, Terry D.
  13. van de Warrenburg, Bart P.
  14. Ristori, Giovanni
  15. Synofzik, Matthis
  16. Perlman, Susan
  17. Schmahmann, Jeremy D.
  18. L’Italien, Gilbert

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1000 Titel
  • Content Validity of the Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) Instrument in Spinocerebellar Ataxia
1000 Autor/in
  1. Potashman, Michele |
  2. Rudell, Katja |
  3. Pavisic, Ivanna |
  4. Suminski, Naomi |
  5. Doma, Rinchen |
  6. Heinrich, Maggie |
  7. Abetz-Webb, Linda |
  8. Beiner, Melissa Wolfe |
  9. Kuo, Sheng-Han |
  10. Rosenthal, Liana S. |
  11. Zesiwicz, Theresa |
  12. Fife, Terry D. |
  13. van de Warrenburg, Bart P. |
  14. Ristori, Giovanni |
  15. Synofzik, Matthis |
  16. Perlman, Susan |
  17. Schmahmann, Jeremy D. |
  18. L’Italien, Gilbert |
1000 Verlag Springer US
1000 Erscheinungsjahr 2024
1000 Publikationstyp
  1. Artikel |
1000 Online veröffentlicht
  • 2024-05-07
1000 Erschienen in
1000 Quellenangabe
  • 23(5):2012-2027
1000 Copyrightjahr
  • 2024
1000 Lizenz
1000 Verlagsversion
  • https://doi.org/10.1007/s12311-024-01700-2 |
  • https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11489265/ |
1000 Publikationsstatus
1000 Begutachtungsstatus
1000 Sprache der Publikation
1000 Abstract/Summary
  • <jats:title>Abstract</jats:title><jats:p>The functional Scale for the Assessment and Rating of Ataxia (f-SARA) assesses Gait, Stance, Sitting, and Speech. It was developed as a potentially clinically meaningful measure of spinocerebellar ataxia (SCA) progression for clinical trial use. Here, we evaluated content validity of the f-SARA. Qualitative interviews were conducted among individuals with SCA1 (<jats:italic>n</jats:italic> = 1) and SCA3 (<jats:italic>n</jats:italic> = 6) and healthcare professionals (HCPs) with SCA expertise (USA, <jats:italic>n</jats:italic> = 5; Europe, <jats:italic>n</jats:italic> = 3). Interviews evaluated symptoms and signs of SCA and relevance of f-SARA concepts for SCA. HCP cognitive debriefing was conducted. Interviews were recorded, transcribed, coded, and analyzed by ATLAS.TI software. Individuals with SCA1 and 3 reported 85 symptoms, signs, and impacts of SCA. All indicated difficulties with walking, stance, balance, speech, fatigue, emotions, and work. All individuals with SCA1 and 3 considered Gait, Stance, and Speech relevant f-SARA concepts; 3 considered Sitting relevant (42.9%). All HCPs considered Gait and Speech relevant; 5 (62.5%) indicated Stance was relevant. Sitting was considered a late-stage disease indicator. Most HCPs suggested inclusion of appendicular items would enhance clinical relevance. Cognitive debriefing supported clarity and comprehension of f-SARA. Maintaining current abilities on f-SARA items for 1 year was considered meaningful for most individuals with SCA1 and 3. All HCPs considered meaningful changes as stability in f-SARA score over 1–2 years, 1–2-point change in total f-SARA score, and deviation from natural history. These results support content validity of f-SARA for assessing SCA disease progression in clinical trials.</jats:p>
1000 Sacherschließung
lokal Female [MeSH]
lokal Disease Progression [MeSH]
lokal Aged [MeSH]
lokal Adult [MeSH]
lokal Humans [MeSH]
lokal Severity of Illness Index [MeSH]
lokal Spinocerebellar Ataxias/physiopathology [MeSH]
lokal Middle Aged [MeSH]
lokal Spinocerebellar ataxia
lokal Cognitive debriefing
lokal Concept elicitation
lokal Male [MeSH]
lokal Spinocerebellar Ataxias/psychology [MeSH]
lokal Reproducibility of Results [MeSH]
lokal Spinocerebellar Ataxias/diagnosis [MeSH]
lokal Research
lokal Gait/physiology [MeSH]
lokal f-SARA
lokal Clinical outcome assessment
1000 Fächerklassifikation (DDC)
1000 Liste der Beteiligten
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  • DeepGreen-ID: 6f883d1fc93c428a934fd1e8176a1d1b ; metadata provieded by: DeepGreen (https://www.oa-deepgreen.de/api/v1/), LIVIVO search scope life sciences (http://z3950.zbmed.de:6210/livivo), Crossref Unified Resource API (https://api.crossref.org/swagger-ui/index.html), to.science.api (https://frl.publisso.de/), ZDB JSON-API (beta) (https://zeitschriftendatenbank.de/api/), lobid - Dateninfrastruktur für Bibliotheken (https://lobid.org/resources/search)
1000 Label
1000 Förderer
  1. This study was supported by Biohaven Pharmaceuticals, Inc. |
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1000 Förderprogramm
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1000 Dateien
1000 Förderung
  1. 1000 joinedFunding-child
    1000 Förderer This study was supported by Biohaven Pharmaceuticals, Inc. |
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    1000 Fördernummer -
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1000 Erstellt am 2025-07-05T18:49:57.763+0200
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1000 Objekt bearb. Mon Aug 11 11:51:28 CEST 2025
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