Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2024 Oct;23(5):2012-2027.
doi: 10.1007/s12311-024-01700-2. Epub 2024 May 7.

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

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

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

Michele Potashman et al. Cerebellum. 2024 Oct.

Abstract

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 (n = 1) and SCA3 (n = 6) and healthcare professionals (HCPs) with SCA expertise (USA, n = 5; Europe, n = 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.

Keywords: Clinical outcome assessment; Cognitive debriefing; Concept elicitation; Spinocerebellar ataxia; f-SARA.

PubMed Disclaimer

Conflict of interest statement

Michele Potashman, Melissa Wolfe Beiner, and Gilbert L’Italien are employed by, and own shares in, Biohaven Pharmaceuticals, Inc. Katja Rudell, Naomi Suminski, Rinchen Doma, and Maggie Heinrich are employees of Parexel and were commissioned by Biohaven Pharmaceuticals, Inc. to conduct the study. Ivanna Pavisic was an employee of Parexel until July 2023 and was commissioned by Biohaven Pharmaceuticals, Inc. to conduct the study. Sheng-Han Kuo, Theresa Zesiwicz, Bart van de Warrenburg, Liana S. Rosenthal, Terry D. Fife, Giovanni Ristori, Matthis Synofzik, and Susan Perlman received consultancy fees from Biohaven Pharmaceuticals, Inc. for participating in the interviews. Linda Abetz-Webb received consultancy fees from Parexel for this study. Sheng-Han Kuo has received consultancy fees from Biohaven Pharmaceuticals, Inc., Praxis Precision Medicines, and Sage Therapeutics. Theresa Zesiwicz has received personal compensation for serving on the advisory boards of Boston Scientific, Reata Pharmaceuticals, and Steminent Biotherapeutics; and received personal compensation as senior editor for Neurodegenerative Disease Management and as a consultant for Steminent Biotherapeutics. Royalties: royalty payments as co-inventor of varenicline for treating imbalance and nonataxic imbalance. Grants: research grant support as Principal Investigator for studies from AbbVie, Biogen, Biohaven Pharmaceuticals, Inc., Boston Scientific, Bukwang Pharmaceuticals Co, Inc., Cala Health, Inc., Cavion, Friedreich’s Ataxia Research Alliance; Houston Methodist Research Institute, National Institutes of Health, REtrotope Inc, and Takeda Development Center Americas, Inc. Bart P. van de Warrenburg has served on advisory boards and/or as consultant for Servier, Vico Therapeutics, Biohaven Pharmaceuticals, Inc., and uniQure. Royalties: BSL—Springer Nature. Grants: Radboud University Medical Center, ZonMw, Gossweiler Foundation, Hersenstichting, NWO, and Christina Foundation. Jeremy D. Schmahmann has served on the editorial board for The Cerebellum, Editorial Board, 1999. Consultancy: Biohaven Pharmaceuticals, Inc. Site Principal Investigator: Biohaven Pharmaceuticals, Inc. clinical trials in ataxia and multiple system atrophy. Research support, commercial entities: Biohaven Pharmaceuticals, Inc. support of clinical trials. Research support, academic entities: National Ataxia Foundation. Research support, foundations, and societies: National Ataxia Foundation, 2019, Principal Investigator license fee payments. Technology or inventions: Brief Ataxia Rating Scale (BARS) and Brief Ataxia Rating Scale revised (BARS2). Copyright held by The General Hospital Corporation. Cerebellar Cognitive Affective/Schmahmann syndrome Scale. Copyright held by The General Hospital Corporation. Patient-Reported Outcome Measure of Ataxia. Copyright held by The General Hospital Corporation. Cerebellar Neuropsychiatric Rating Scale. Copyright held by The General Hospital Corporation.

Figures

Fig. 1
Fig. 1
Study design and development of the conceptual framework Abbreviations: f-SARA modified functional Scale for the Assessment and Rating of Ataxia, HCP healthcare professional, SCA spinocerebellar ataxia
Fig. 2
Fig. 2
Overarching concepts identified in interviews with individuals with SCA Abbreviation: SCA spinocerebellar ataxia
See this image and copyright information in PMC

References

    1. Klockgether T, Mariotti C, Paulson HL. Spinocerebellar ataxia. Nat Rev Dis Primers. 2019;5:24. 10.1038/s41572-019-0074-3 - PubMed
    1. Diallo A, Jacobi H, Tezenas du Montcel S, Klockgether T. Natural history of most common spinocerebellar ataxia: a systematic review and meta-analysis. J Neurol. 2021;268:2749–56. 10.1007/s00415-020-09815-2 - PubMed
    1. Yap KH, Azmin S, Che Hamzah J, Ahmad N, van de Warrenburg B, Mohamed Ibrahim N. Pharmacological and non-pharmacological management of spinocerebellar ataxia: a systematic review. J Neurol. 2022;269:2315–37. 10.1007/s00415-021-10874-2 - PubMed
    1. Hengel H, Pellerin D, Wilke C, Fleszar Z, Brais B, Haack T, et al. As frequent as polyglutamine spinocerebellar ataxias: SCA27B in a large German autosomal dominant ataxia cohort. Mov Disord. 2023;38:1557–8. 10.1002/mds.29559 - PubMed
    1. Wilke C, Pellerin D, Mengel D, Traschutz A, Danzi MC, Dicaire MJ, et al. GAA-FGF14 ataxia (SCA27B): phenotypic profile, natural history progression and 4-aminopyridine treatment response. Brain. 2023;146:4144–57. 10.1093/brain/awad157 - PubMed