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

描述（由申请人提供）：如何将患者对幸福的看法科学地纳入癌症研究？关于PRO措施相对优点的详细信息很少，这使得临床研究人员只能猜测选择哪种措施以及何时最好地使用它们。这项R01研究的总体目标是证明最近开发的、简单的、简短的患者报告的幸福感和症状困扰评估是心理测量学上兼容的，并且是癌症临床研究中实用的预后生物标志物。具体来说，我们将比较美国国立卫生研究院资助的患者报告结果测量信息系统（PROMIS）简短表格与基于pro版本的不良事件通用术语标准（CTCAE）或PRO-CTCAE，以及简单的单项症状线性模拟自我评估（LASA）措施。我们将实施一个多站点的主协议，在四年的时间里，从梅奥诊所癌症中心、安德森癌症中心和Memorial-Sloan-Kettering癌症中心收集1800名癌症患者。PRO数据将在每个患者的三个观察点收集，大约间隔一个月，在基线和纵向上测试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，平均每位患者随访2年。我们将测试数据收集方式（纸质与电子）的效果。本研究将提供一个完整的PROMIS测量相对于新的PRO-CTCAE测量和简单的单项数值类似物在癌症患者中的心理测量概况，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。虽然这项提议的重点是癌症，但这项研究也与其他疾病有关。具体目标1：比较PROMIS、PRO-CTCAE和其他简单的、患者报告的测量方法的心理测量特性，为癌症临床研究中的患者福祉和安全产生标准化的pro。将进行广泛的心理测量测试，以评估PROMIS和PRO-CTCAE工作之间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2：测试PROMIS和其他简单的、患者报告的预后指标，以评估总生存期（OS）、无病生存期（DFS）和无进展生存期（PFS）的预后能力。本研究的目的是评估PROMIS、PRO-CTCAE和LASA指标对OS、DFS和PFS在多个疾病部位和分期的独立预后能力。具体目标3：开发和测试在临床环境中收集PROMIS和其他简单的、患者报告的结果测量的新策略。本目的目的是评估在临床环境中上述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度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测量。

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. PUBLIC HEALTH RELEVANCE: The purpose of this project is to compare three different questionnaires that are designed to evaluate patient well-being, especially for people with cancer. The project will also test different ways of collecting questionnaire data such as pencil-and-paper forms, computerized forms, and automated telephone surveys.