Biohaven Pharmaceuticals, Inc, New Haven, CT, USA.
COA Science, Parexel International, London, UK.
Cerebellum. 2024 Oct;23(5):2012-2027. doi: 10.1007/s12311-024-01700-2. Epub 2024 May 7.
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.
功能性共济失调评定量表（f-SARA）评估步态、站位、坐姿和言语。它是作为一种潜在的有临床意义的脊髓小脑共济失调（SCA）进展的衡量标准而开发的，用于临床试验。在这里，我们评估了 f-SARA 的内容效度。在 SCA1（n=1）和 SCA3（n=6）患者以及具有 SCA 专业知识的医疗保健专业人员（HCP）（美国，n=5；欧洲，n=3）中进行了定性访谈。访谈评估了 SCA 的症状和体征以及 f-SARA 概念对 SCA 的相关性。对 HCP 进行认知性内容审核。对访谈进行了记录、转录、编码和 ATLAS.TI 软件分析。SCA1 和 3 的患者报告了 85 种 SCA 的症状、体征和影响。所有人都表示行走、站立、平衡、言语、疲劳、情绪和工作困难。所有 SCA1 和 3 的患者均认为步态、站位和言语是 f-SARA 的相关概念；3 人认为坐姿（42.9%）是相关的。所有 HCP 均认为步态和言语是相关的；5 人（62.5%）表示站位也是相关的。坐姿被认为是疾病晚期的指标。大多数 HCP 建议纳入肢体项目将增强临床相关性。认知性内容审核支持了 f-SARA 的清晰性和可理解性。对于大多数 SCA1 和 3 的患者来说，在 f-SARA 项目上保持 1 年的现有能力被认为是有意义的。所有 HCP 都认为有意义的变化是 f-SARA 评分在 1-2 年内保持稳定，总 f-SARA 评分变化 1-2 分，以及偏离自然病程。这些结果支持了 f-SARA 在临床试验中评估 SCA 疾病进展的内容效度。
