Validating Assessments of Real-World Outcome

验证对现实世界结果的评估

• 批准号: 7643386
• 负责人: Thomas L. Patterson
• 金额: $ 34.76万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-09-25 至 2012-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7643386
• 关键词: Activities of Daily Living; Adopted; Affect; Age; Attention; Awareness; Caregivers; Case Manager; Classification; Cognition; Cognitive; Cognitive remediation; Companions; Data Analyses; Demographic Factors; Education; Environment; Eye; Future; Generations; Goals; HIV Infections; Hand; Impaired cognition; Impairment; Life; Measures; Medical; Monitor; Neurocognitive; Neuropsychological Tests; Occupational; Onset of illness; Outcome; Outcome Assessment; Outcome Measure; Patient Self-Report; Patients; Performance; Pharmacology; Quality of life; Relative (related person); Reporter; Reporting; Research; Research Personnel; Rest; Sampling; Schizophrenia; Self Care; Series; Site; Symptoms; Techniques; Test Result; Testing; Time; United States National Institutes of Health; Validation; Validity of Self Report; base; cost; depression; depressive symptoms; design; disability; functional disability; functional outcomes; functional status; improved; informant; longitudinal design; neuropsychological; programs; psychosocial; response; skills; social; tool; treatment trial

DESCRIPTION (provided by applicant): This collaborative study (the P.I. of whose companion application is Dr. Philip D. Harvey) aims to validate assessments of real-world (RW) functional outcomes in people with schizophrenia. Schizophrenia patients are often impaired in their social and occupational functioning, their self-care, and their ability to live independently. Not only do these impairments worsen the patient's quality of life, they also increase the societal cost of his or her illness. While these impairments are evident in most patients, surprisingly little attention has been paid to the validity of assessments of these outcomes. Since the current tool of choice in predicting functional disability in schizophrenia is neuropsychological (NP) testing, proposals to reduce disability have tended to focus on short-term treatment of cognitive impairments. However, such trials are not well suited either to induce or identify improvements in real-world functioning. Some researchers have proposed switching to measures of functional capacity instead, but recent evidence suggests that the correlations obtained between functional-capacity measures, NP test results, and real-world outcomes are largely a function of the real-world rating scale that is used. Thus systematic studies are needed to determine the optimal means for measuring real-world disability and its response to treatment. The present proposal has three distinct goals. It aims to determine: (1) the most accurate and practical scale for rating real-world outcomes in schizophrenia; (2) the optimal user of the scale; and (3) the factors that may impair the validity of self-reports of real-world outcomes on the part of schizophrenia patients. We adopt a construct-validation approach, using NP testing and functional-capacity measures as indicators of the construct and examining the convergence of various real-world outcome measures with these two indicators. Sophisticated data analysis will be performed to determine the convergences, and retesting will be performed to determine the test-retest stability of the candidate measures. The overall goal is to identify improved strategies for measuring real-world disability in schizophrenia and to inform the coming generation of treatment trials aimed at cognitive and functional enhancement through pharmacology and cognitive remediation in terms of the most suitable outcomes measures.

描述（由申请人提供）：这项合作研究（P.I.菲利普·D博士是他的伴侣。Harvey）旨在验证精神分裂症患者真实世界（RW）功能结局的评估。精神分裂症患者的社会和职业功能、自我照顾和独立生活能力往往受损。这些损伤不仅使患者的生活质量恶化，还增加了他或她的疾病的社会成本。虽然这些损害在大多数患者中是明显的，但令人惊讶的是，很少有人关注这些结果评估的有效性。由于目前预测精神分裂症功能障碍的首选工具是神经心理学（NP）测试，因此减少残疾的建议往往侧重于认知障碍的短期治疗。然而，这些试验并不适合诱导或识别现实世界功能的改善。一些研究人员建议改用功能能力的测量，但最近的证据表明，功能能力测量，NP测试结果和现实世界的结果之间的相关性在很大程度上是所使用的现实世界的评级量表的函数。因此，需要进行系统的研究，以确定衡量现实世界残疾及其对治疗的反应的最佳手段。本提案有三个明确的目标。其目的是确定：（1）评定精神分裂症真实世界结局的最准确和实用的量表;（2）量表的最佳使用者;（3）可能损害精神分裂症患者自我报告真实世界结局有效性的因素。我们采用了一种结构验证方法，使用NP测试和功能能力指标作为结构的指标，并检查各种现实世界的结果指标与这两个指标的收敛。将进行复杂的数据分析，以确定收敛性，并进行重新检验，以确定候选指标的重测稳定性。总体目标是确定用于测量精神分裂症患者的真实残疾的改进策略，并为下一代治疗试验提供信息，这些试验旨在通过药理学和认知补救措施来增强认知和功能。