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

描述（由申请人提供）：如何将患者的健康观点科学地纳入癌症研究？详细介绍PRO措施相对优点的信息很少，让临床研究人员猜测选择哪些措施以及何时最好地部署它们。这项R 01研究的总体目标是证明，最近开发的，简单的，简短的评估患者报告的幸福和症状困扰是心理测量兼容和实用的预后生物标志物癌症临床研究。具体而言，我们将比较NIH资助的患者报告结局测量信息系统（PROMIS）简表与基于PRO的不良事件通用术语标准（CTCAE）或PRO-CTCAE版本，以及简单的单项症状线性模拟自我评估（LASA）措施。我们将实施一个多中心的主方案，在四年内从马约诊所癌症中心、医学博士和国家癌症中心收集1800名癌症患者。安德森癌症中心和纪念斯隆凯特琳癌症中心。将在每例患者的三个观察点收集PRO数据，大约间隔一个月，以测试基线和纵向的心理测量和预后特性。将对患者进行总生存期（OS）、无病生存期（DFS）和无进展生存期（PFS）随访，每例患者平均随访2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PROMIS指标相对于新PRO-CTCAE指标和简单单项数值模拟在癌症患者中的完整心理测量学特征，并评估它们是否可作为癌症临床研究的实用预后生物标志物。虽然这项建议的重点是癌症，但这项调查也与其他疾病有关。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指标的心理测量特性，以生成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，以评价PROMIS和PRO-CTCAE工作相对于简单的单项评估的有效性、反应性、冗余性和兼容性。具体目标二：测试PROMIS和其他简单的、患者报告的结局指标对总生存期（OS）、无病生存期（DFS）和无进展生存期（PFS）的预后能力。本研究旨在评估PROMIS、PRO-CTCAE和LASA指标在多个疾病部位和分期中对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结局指标的新策略。本目标旨在评价临床环境中上述PRO措施的各种数据收集模式的相对优点。我们将测试患者的接受性和测量误差基于纸张，基于计算机和交互式语音应答（IVR）版本的PRO措施。

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.