Validation of Measures of real-world outcome

描述(由申请人提供)：这项合作研究旨在验证对精神分裂症患者的真实世界(RW)功能结果的评估。精神分裂症患者的社会和职业功能、自理能力和独立生活能力往往受到损害。这些损伤不仅会恶化患者的生活质量，还会增加他或她的疾病的社会成本。虽然这些损害在大多数患者中都很明显，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患者功能障碍的首选工具是神经心理学(NP)测试，减少残疾的建议往往侧重于认知障碍的短期治疗。然而，这样的试验既不适合诱导或识别真实世界功能的改善。一些研究人员建议改用功能能力测量方法，但最近的证据表明，功能能力测量方法、NP测试结果和现实世界结果之间的相关性在很大程度上是所使用的真实世界评级标准的函数。因此，需要进行系统研究，以确定衡量现实世界残疾及其对治疗反应的最佳手段。目前的提议有三个不同的目标。它的目的是确定：(1)最准确和最实用的精神分裂症结果分级量表；(2)该量表的最佳使用者；(3)可能影响精神分裂症患者自我报告现实世界结果有效性的因素。我们采用结构验证方法，使用NP测试和功能能力度量作为结构的指标，并检查各种现实世界结果衡量标准与这两个指标的趋同。将进行复杂的数据分析以确定收敛，并将进行重新测试以确定候选测量的重测稳定性。总的目标是确定衡量精神分裂症真实世界残疾的改进战略，并向下一代治疗试验提供信息，旨在通过药理学和认知补救来增强认知和功能，以获得最合适的结果衡量标准。

DESCRIPTION (provided by applicant): This collaborative study (the P.I. of whose companion application is Dr. Thomas Patterson) 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.