Understanding patient-reported outcome measures in Huntington disease: at what point is cognitive impairment related to poor measurement reliability?
Understanding patient-reported outcome measures in Huntington disease: at what point is cognitive impairment related to poor measurement reliability?
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DOI：
10.1007/s11136-018-1912-6
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发表时间：
2018-10-01
影响因子：
3.5
通讯作者：
Stout, J. C.
中科院分区：
文献类型：
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作者：
Carlozzi, N. E.;Schilling, S.;Stout, J. C.
目的 亨廷顿病 (HD) 的症状进展与认知能力下降有关，这可能会干扰症状的自我报告。不幸的是，随着 HD 进展，支持或反驳患者报告结果 (PRO) 心理测量可靠性的数据有限。鉴于 PRO 越来越被认为是衡量新疗法疗效的重要指标，这是有问题的。 方法 我们检查了 509 名 HD 前期、早期或晚期患者的 HDQLIFE 测量系统（言语困难、吞咽困难、舞蹈病）的 PRO 数据。还收集了临床医生对运动功能的评估（来自 UHDRS 的项目）和标准化客观认知评估（Stroop、符号数字模态）。我们使用不同项目功能 (DIF) 来检查 HD 阶段（预显期、早期、晚期）的项目偏差以及相对于认知表现的项目偏差。我们还检查了自我报告和临床医生评分之间的相关性。使用考虑总体认知能力的回归模型来确定 PRO 的心理测量可靠性。结果 大多数 PRO 项目在分期和认知方面均不受 DIF 影响。 PRO 和临床医生报告之间存在适度的相关性（范围从 -0.40 到 -0.60）。模型分析表明，心理测量的可靠性随着认知能力的下降和疾病阶段的进展而降低；当舞蹈病得分 < 136、言语困难得分 < 109 和吞咽困难得分 < 179 时，分半可靠性会受到损害（即分半可靠性 < 0.80）。 结论 结果表明，随着 HD 症状进展和认知能力下降，PRO 的心理测量可靠性可能会受到损害。当总认知得分超过临界阈值时，临床医生应考虑将 PRO 与其他类型的评估结合起来。
Purpose Symptom progression in Huntington disease (HD) is associated with cognitive decline which may interfere with the self-report of symptoms. Unfortunately, data to support or refute the psychometric reliability of patient-reported outcomes (PROs) as HD progresses are limited. This is problematic given that PROs are increasingly recognized as important measures of efficacy for new treatments.Methods We examined PRO data from the HDQLIFE Measurement System (Speech Difficulties; Swallowing Difficulties; Chorea) in 509 individuals with premanifest, early-stage, or late-stage HD. Clinician-administered assessments of motor functioning (items from the UHDRS) and standardized objective assessments of cognition (Stroop, Symbol Digit Modalities) were also collected. We examined item bias using differential item functioning (DIF) across HD stage (premanifest, early-, late-) and relative to cognitive performance. We also examined the correlations between self-report and clinician ratings. Regression models that considered total cognitive ability were utilized to determine psychometric reliability of the PROs.Results Most PRO items were free from DIF for both staging and cognition. There were modest correlations between PROs and clinician report (ranged from -0.40 to -0.60). Modeling analyses indicated that psychometric reliability breaks down with poorer cognition and more progressed disease stage; split-half reliability was compromised (i.e., split-half reliability < 0.80) when scores were < 136 for Chorea, < 109 for Speech Difficulties, and < 179 for Swallowing Difficulties.Conclusions Results indicate that the psychometric reliability of PROs can be compromised as HD symptoms progress and cognition declines. Clinicians should consider PROs in conjunction with other types of assessments when total cognition scores exceed critical thresholds.