Sage Therapeutics, Inc., Cambridge, MA, USA.
Modus Outcomes, a Division of THREAD, St. James Square, Cheltenham, England, UK.
J Huntingtons Dis. 2024;13(3):385-397. doi: 10.3233/JHD-240001.
BACKGROUND: The Huntington's Disease (HD) Everyday Functioning (Hi-DEF) is a new patient-reported outcome (PRO) instrument designed to measure the impact of cognitive impairment on daily functioning in the early stages of HD. OBJECTIVE: To assess the measurement properties and finalize item content of the Hi-DEF. METHODS: A cross-sectional, observational psychometric validation study was conducted among individuals with early stages of HD at 9 US centers of excellence. Rasch Measurement Theory (RMT) analysis of the initial draft version of the Hi-DEF (47 items) and subscales (i.e., 'Home', 'At work', 'Driving', and 'Communication') was conducted to examine measurement properties including sample-to-scale targeting, suitability of response scale (ordering of response thresholds), scale cohesiveness (item fit), local independence, and person fit. RESULTS: 151 participants (mean age 47 years (SD 12), 59% female) were included. Seven items were removed based on dependency and item fit. The remaining 40-item version of the Hi-DEF demonstrated good measurement properties. Across the four subscales, targeting ranged from 49-70% (72% full scale), reliability ascertained by person separation index ranged from 0.53-0.87 (0.92 full scale), response scales were ordered for 25-100% of items (75% full scale), 0-12% items displayed misfit (2% full scale), and 0-1% (2% full scale) item pairs displayed dependency. CONCLUSIONS: Our study supports the psychometric integrity of the Hi-DEF as a reliable and valid new PRO instrument designed to assess the impact of cognitive impairment on daily functioning in the early stages of HD. Future work will evaluate the external validity and utility in clinical trial applications.
背景:亨廷顿病(HD)日常生活功能(Hi-DEF)是一种新的患者报告结局(PRO)工具,旨在测量认知障碍对 HD 早期日常生活功能的影响。
目的:评估 Hi-DEF 的测量特性并确定其项目内容。
方法:在 9 个美国卓越中心进行了一项横断面、观察性心理测量验证研究,研究对象为 HD 早期阶段的个体。采用 Rasch 测量理论(RMT)对 Hi-DEF(47 项)的初始草案版本和子量表(即“家庭”、“工作”、“驾驶”和“沟通”)进行分析,以检查测量特性,包括样本与量表的目标值、反应量表的适宜性(反应阈值的顺序)、量表的一致性(项目拟合)、局部独立性和个体拟合。
结果:纳入 151 名参与者(平均年龄 47 岁(标准差 12 岁),59%为女性)。基于依存性和项目拟合,有 7 项被删除。剩余的 40 项 Hi-DEF 版本具有良好的测量特性。在四个子量表中,目标值范围为 49-70%(72%的总分),个体分离指数确定的可靠性范围为 0.53-0.87(0.92 的总分),反应量表对 25-100%的项目进行排序(75%的总分),0-12%的项目存在不拟合(2%的总分),0-1%(2%的总分)的项目对存在依存性。
结论:我们的研究支持 Hi-DEF 的心理测量完整性,它是一种可靠且有效的新 PRO 工具,旨在评估认知障碍对 HD 早期日常生活功能的影响。未来的工作将评估其在临床试验应用中的外部有效性和实用性。
J Int Neuropsychol Soc. 2023-5
Front Aging Neurosci. 2021-8-3
Tremor Other Hyperkinet Mov (N Y). 2020-7-9
J Patient Rep Outcomes. 2019-7-30
Eur J Neurol. 2017-9-22