Validation of Measures of real-world outcome

描述（由申请人提供）：这项合作研究（其同伴申请的P.I.是Thomas Patterson博士）旨在验证精神分裂症患者真实世界（RW）功能结果的评估。精神分裂症患者的社会和职业功能、自我保健和独立生活能力往往受损。这些损害不仅使病人的生活质量恶化，还增加了他或她的疾病的社会成本。虽然这些损伤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功能性残疾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，这样的试验并不适合于诱导或确定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，但是，最近的证据表明，功能能力测量、NP测试结果和现实世界结果之间的相关性在很大程度上取决于所使用的现实世界评定量表。因此，需要系统的研究来确定衡量现实世界残疾及其对治疗的反应的最佳方法。目前的提议有三个明确的目标。它的目的是确定：(1)最准确和实用的评估精神分裂症现实结果的量表；(2)最优用户的规模；(3)影响精神分裂症患者现实生活结果自我报告效度的因素。我们采用构造验证方法，使用NP测试和功能能力测量作为构造的指标，并使用这两个指标检查各种现实世界结果测量的收敛性。将执行复杂的数据分析以确定收敛性，并将执行重新测试以确定候选度量的测试-重新测试稳定性。总体目标是确定衡量精神分裂症现实世界残疾的改进策略，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来增强认知和功能，以获得最合适的结果测量。

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.