Assessing PROMIS and other simple patient-reported measures for cancer research

描述(由申请人提供)：如何将患者的幸福观科学地纳入癌症研究？几乎没有详细说明PRO措施相对优点的信息，这让临床研究人员猜测应该选择哪些措施，以及何时部署它们是最佳的。这项R01研究的总体目标是证明，最近开发的对患者报告的幸福感和症状痛苦的简单、简短的评估是癌症临床研究的心理测量学兼容和实用的预后生物标记物。具体地说，我们将比较NIH资助的患者报告结果衡量信息系统(PROIS)缩写与不良事件通用术语标准(CTCAE)或PRO-CTCAE的PRO版本，以及简单的单项症状线性模拟自我评估(LASA)措施。我们将实施一项多地点主方案，在四年内从梅奥诊所癌症中心、M.D.安德森癌症中心和纪念-斯隆-凯特琳癌症中心接收1800名癌症患者。PRO数据将在每个患者的三个观察点收集，大约相隔一个月，以测试基线和纵向的心理测量和预后特性。患者将接受总生存期(OS)、无病生存期(DFS)和无进展生存期(PFS)的随访，每个患者平均随访两年。我们将测试数据收集模式(纸质与电子)的效果。本研究将提供癌症患者PROMIS测量相对于新的PROCTCAE测量方法和简单的单项数值模拟的完整的心理测量学概况，并评估它们是否可以作为癌症临床研究的实用预后生物标志物。虽然这项提案的重点是癌症，但这项调查也与其他疾病相关。将追求三个具体目标：具体目标1：比较PROMIS、PRO-CTCAE和其他简单的、患者报告的措施的心理测量学特性，以产生癌症临床研究中患者福祉和安全性的标准化PRO。将进行广泛的心理测量测试，以评估PROMIS和PROCTCAE努力之间的有效性、响应性、冗余性和兼容性，而不是简单的单项评估。具体目标2：测试PROMIS和其他简单的、患者报告的结果指标，以确定总体生存(OS)、无病生存(DFS)和无进展生存(PFS)的预后能力。本研究的目的是评估PROMIS、PRO-CTCAE和LASA方法对多个疾病部位和阶段的OS、DFS和PFS的独立预后能力。具体目标3：在临床环境中开发和测试收集Promis和其他简单的、患者报告的结果测量的新策略。其目的是评估各种数据收集模式在临床环境中对上述PRO措施的相对优劣。我们将测试PRO措施的纸质、计算机和交互式语音应答(IVR)版本的患者接受性和测量误差。

DESCRIPTION (provided by applicant): How can the patient's perspective of well-being be incorporated scientifically into cancer research? Little information detailing the relative merits of PRO measures exists, leaving clinical researchers guessing which measures to choose and when best to deploy them. The overall goal of this R01 investigation is to demonstrate that recently-developed, simple, brief assessments of patient-reported well-being and symptom distress are psychometrically compatible and practical prognostic biomarkers for cancer clinical research. Specifically, we will compare the NIH-funded Patient-Reported Outcome Measurement Information System (PROMIS) short forms with the PRO-based version of the Common Terminology Criteria for Adverse Events (CTCAE) or PRO-CTCAE, and simple, single-item symptom linear analogue self-assessment (LASA) measures. We will implement a multi-site master protocol that will accrue 1800 cancer patients over four years from the Mayo Clinic Cancer Center, the M.D. Anderson Cancer Center, and the Memorial-Sloan-Kettering Cancer Center. PRO data will be collected at three observation points per patient, roughly one month apart to test psychometric and prognostic properties at baseline and longitudinally. Patients will be followed for overall survival (OS), disease-free survival (DFS), and progression-free survival (PFS) with an average followup per patient of two years. We will test the effect of data collection mode (paper versus electronic). This research will provide a complete psychometric profile of the PROMIS measures relative to the new PRO-CTCAE measures and simple single-item numerical analogues in cancer patients and assess whether they could serve as practical prognostic biomarkers for cancer clinical research. Although the focus of this proposal is on cancer, this investigation is relevant to other conditions as well. Three specific aims will be pursued: Specific Aim 1: Compare psychometric properties of PROMIS, PRO-CTCAE, and other simple, patient- reported measures to produce standardised PROs for patient well-being and safety in cancer clinical research. Extensive psychometric testing will be carried out to evaluate the validity, responsiveness, redundancy, and compatibility between the PROMIS and PRO-CTCAE efforts relative to simple, single item assessments. Specific Aim 2: Test the PROMIS and other simple, patient-reported outcome measures for prognostic capability for overall survival (OS), disease-free survival (DFS), and progression-free survival (PFS). The purpose of this aim is to estimate the independent prognostic ability of the PROMIS, PRO-CTCAE, and LASA measures for OS, DFS, and PFS across multiple disease sites and stages. Specific Aim 3: Develop and test new strategies for collecting PROMIS and other simple, patient- reported outcome measures in clinical settings. The purpose of this aim is to evaluate the relative merits of various data collection modes for the aforementioned PRO measures in a clinic setting. We will test patient receptivity and measurement error paper-based, computer-based, and interactive voice response (IVR) versions of the PRO measures.