Ataxia Center, Cognitive Behavioral Neurology Unit, Laboratory for Neuroanatomy and Cerebellar Neurobiology, Massachusetts General Hospital and Harvard Medical School, Boston, Massachusetts, USA.
Global Health Outcomes and Epidemiology, Biohaven Pharmaceuticals, New Haven, Connecticut, USA.
Mov Disord. 2021 Oct;36(10):2367-2377. doi: 10.1002/mds.28670. Epub 2021 Jun 11.
BACKGROUND: Assessment of cerebellar ataxia has been confined to rating scales, gait laboratories, and wearable sensors agnostic to patient input. OBJECTIVES: The objective of this study was to develop a Patient-Reported Outcome Measure of Ataxia. METHODS: (1) The conceptual framework, item pool development, and domain selection were developed using online surveys completed by 147 ataxia patients. Responses generated the 70-item Patient-Reported Outcome Measure of Ataxia, scored on a 0-4 Likert scale. (2) Cognitive debrief in 17 patients grouped by ataxia severity assessed content validity, readability, and comprehension. (3) Psychometric validation by 78 anonymized ataxia patients included test-retest reliability, responsiveness to ataxia severity, internal consistency (Cronbach's alpha), and item-total score correlations. (4) Validation was tested against measures of ataxia and quality of life in 20 patients. (5) Items were rank-ordered to develop the Patient-Reported Outcome Measure of Ataxia Short Form. RESULTS: Three thousand eight hundred fifty-five symptoms were grouped into 3 domains (physical, activities of daily living, mental health) and 14 subdomains. The Patient-Reported Outcome Measure of Ataxia was comprehensible, important, and relevant. Internal consistency, reliability, and test-retest reliability were high. Scores were responsive to ataxia severity stages 1, 2, and 3: mean ± standard deviation 81.0 ± 37.0, 129.6 ± 32.0, and 151.1 ± 41.3, respectively (r = 0.58, P < 0.0001). The Patient-Reported Outcome Measure of Ataxia was validated against measures of motor ataxia, quality of life, and mental health. It had an R of 0.82 (P < 0.0001) with the preliminary Patient-Reported Outcome Measure of Ataxia Short Form. CONCLUSIONS: The Patient-Reported Outcome Measure of Ataxia is valid and reliable in cerebellar ataxia patients. It has the potential to improve patient care and natural history studies and quantify the efficacy of novel therapeutics in clinical trials. © 2021 International Parkinson and Movement Disorder Society.
背景：对小脑性共济失调的评估一直局限于评分量表、步态实验室和对患者输入无感知的可穿戴传感器。 目的：本研究的目的是开发一种基于患者报告的共济失调结局测量工具。 方法：（1）使用在线调查，由 147 名共济失调患者完成，制定了概念框架、项目池开发和领域选择。基于调查结果生成了 70 项的基于患者报告的共济失调结局测量工具，采用 0-4 级 Likert 量表评分。（2）通过将 17 名共济失调患者按严重程度分组进行认知性澄清，评估内容有效性、可理解性和理解度。（3）对 78 名匿名共济失调患者进行心理测量学验证，包括重测信度、对共济失调严重程度的反应性、内部一致性（Cronbach's alpha）和项目总分相关性。（4）在 20 名患者中对其进行验证，评估其与共济失调和生活质量的测量工具的相关性。（5）对项目进行排序，以开发基于患者报告的共济失调结局测量工具简短版。 结果：3855 个症状被分为 3 个领域（身体、日常生活活动、心理健康）和 14 个亚领域。基于患者报告的共济失调结局测量工具是可以理解的、重要的和相关的。内部一致性、可靠性和重测信度都很高。评分对 1、2 和 3 阶段的共济失调严重程度有反应：平均值±标准差分别为 81.0±37.0、129.6±32.0 和 151.1±41.3（r=0.58，P<0.0001）。基于患者报告的共济失调结局测量工具与运动性共济失调、生活质量和心理健康的测量工具具有相关性。与初步的基于患者报告的共济失调结局测量工具简短版相比，其 R 值为 0.82（P<0.0001）。 结论：基于患者报告的共济失调结局测量工具在小脑性共济失调患者中是有效且可靠的。它具有改善患者护理和自然史研究以及量化临床试验中新型治疗药物疗效的潜力。© 2021 国际帕金森病和运动障碍学会。
Mov Disord. 2021-10
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