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

Michele Potashman, Katja Rudell, Ivanna Pavisic, Naomi Suminski, Rinchen Doma, Maggie Heinrich, Linda Abetz-Webb, Melissa Wolfe Beiner,Sheng-Han Kuo,Liana S. Rosenthal, Theresa Zesiwicz, Terry D. Fife,Bart P. van de Warrenburg,Giovanni Ristori,Matthis Synofzik,Susan Perlman, Jeremy D. Schmahmann, Gilbert L’Italien

The Cerebellum(2024)

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摘要
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
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关键词
Spinocerebellar ataxia,f-SARA,Clinical outcome assessment,Concept elicitation,Cognitive debriefing
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