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.
摘要：
共济失调评估和评级功能量表（f-SARA）评估步态，Stance,坐着，和演讲。它被开发为用于临床试验的脊髓小脑共济失调（SCA）进展的潜在临床意义的量度。这里,我们评估了f-SARA的内容效度。对SCA1（n=1）和SCA3（n=6）的个体以及具有SCA专业知识的医疗保健专业人员（HCP）进行了定性访谈（美国，n=5；欧洲，n=3)。访谈评估了SCA的症状和体征以及f-SARA概念与SCA的相关性。进行HCP认知汇报。采访被记录下来，转录，编码,并由ATLAS分析。TI软件。患有SCA1和3的人报告了85种症状，标志,以及SCA的影响。所有都表明行走困难，立场，balance,演讲,疲劳,情感,和工作。所有SCA1和3的人都考虑步态，Stance,和语音相关的f-SARA概念；3认为坐姿相关(42.9%)。所有HCP均认为步态和言语相关；5（62.5％）表示姿态相关。坐姿被认为是晚期疾病指标。大多数HCP建议纳入阑尾项目将增强临床相关性。认知汇报支持f-SARA的清晰和理解。对于大多数患有SCA1和3的人来说，保持f-SARA项目的当前能力1年被认为是有意义的。所有HCP认为有意义的变化是f-SARA评分在1-2年内的稳定性，f-SARA总分变化1-2分,与自然史的偏离。这些结果支持f-SARA在临床试验中评估SCA疾病进展的内容有效性。
