Adaption and Validation of PROMIS for use in Vasculitis

PROMIS 用于血管炎的调整和验证

• 批准号: 8461762
• 负责人: Peter A Merkel
• 金额: $ 41.66万
• 依托单位: UNIVERSITY OF PENNSYLVANIA
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-09-15 至 2016-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8461762
• 关键词: Antineutrophil Cytoplasmic Antibodies; Boxing; Churg-Strauss Syndrome; Clinical Research; Clinical Trials; Computers; Cross-Sectional Studies; Data; Development; Disease; Effectiveness; Fatigue; Goals; Health; Individual; Inflammation; Information Systems; Institutes; Ischemia; Lead; Longitudinal Studies; Measurement; Measures; Methods; Microscopic polyangiitis; Mission; North America; Organ; Outcome; Outcome Measure; Patient Outcomes Assessments; Patients; Physicians; Population; Process; Questionnaires; Randomized; Rare Diseases; Registries; Relative (related person); Reporting; Request for Applications; Research; Research Infrastructure; Research Personnel; Research Project Grants; Resources; SF-36; Sample Size; Symptoms; System; Testing; Therapeutic Agents; Therapeutic Clinical Trial; Time; United States National Institutes of Health; Validation; Vasculitis; Work; base; burden of illness; cohort; comparative effectiveness; effectiveness trial; health related quality of life; highly advanced system; impression; improved; instrument; novel; primary outcome; research clinical testing; standard measure; tool

DESCRIPTION (provided by applicant): The overarching goal of the proposed work is to more effectively incorporate patients' perspectives into the assessment of disease activity and burden among patients with ANCA-associated vasculitis [AAV, granulomatosis with polyangiitis (Wegener's), microscopic polyangiitis, and Churg-Strauss syndrome]. This project seeks to demonstrate the validity and feasibility of incorporating the Patient Reported Outcomes Measurement System (PROMIS) into clinical research in AAV. This project has five specific aims: Specific Aim 1: Determine the construct validity of PROMIS instruments in AAV through cross-sectional analysis of subjects within the Vasculitis Clinical Research Consortium (VCRC) patient cohort, utilizing other HRQOL instruments and physician-determined measures of vasculitis disease activity and damage as constructs. Specific Aim 2: Determine the longitudinal validity, responsiveness, and clinically important difference for the PROMIS instruments in an AAV population by comparing changes over time to the clinical evaluation, fatigue SF-36, and a patient global impression of change measure. Specific Aim 3: Determine the additive/complementary value of PROMIS assessments in defining the disease beyond standard measures of HRQOL and disease activity in AAV. Specific Aim 4: Use selected PROMIS instruments in a randomized therapeutic clinical trial in AAV. Specific Aim 5: Establish a "tool box" of PROMIS symptomatic measures that are the most useful for clinical research in AAV by assessing i) patients' ratings of the importance of the instrument domains; ii) the usefulness in describing the extent of the disease process; and iii) the relative efficiency and feasibility of the use of various PROMIS instruments in an RCT; and then providing researchers with informed guidance to integrate PROMIS into clinical investigation of AAV. The study will be conducted by the Vasculitis Clinical Research Consortium (VCRC), the largest collaborative clinical research infrastructure in North America and is fully responsive to the NIH RFA for PROMIS by utilizing already-established research cohorts and resources, including i) the VCRC Longitudinal Study cohorts for AAV; ii) the RITAZAREM clinical trial in AAV; and iii) the on-line VCRC Patient Contact Registry. PUBLIC HEALTH RELEVANCE: The proposed project will explore novel methods to incorporate how patients with vasculitis feel about the burden of their disease into clinical research in order to both better understand the disease and to help develop a set of research tools to measure the disease from the patients' perspective. The project will test the Patient Reported Outcomes Measurement System (PROMIS), a highly advanced system developed by the NIH to measure patients' ratings of disease-related health problems. The research will produce important advances to provide a method to incorporate patients' own assessments of their disease into research. This project is fully relevant to the mission and goals of the Nationa Institutes of Health.

描述（由申请人提供）：拟议工作的总体目标是更有效地将患者的观点纳入对 ANCA 相关血管炎 [AAV、肉芽肿性多血管炎（韦格纳氏）、显微镜下多血管炎、和 Churg-Strauss 综合征]。该项目旨在证明将患者报告结果测量系统 (PROMIS) 纳入 AAV 临床研究的有效性和可行性。该项目有五个具体目标： 具体目标 1：通过对血管炎临床研究联盟 (VCRC) 患者队列中的受试者进行横断面分析，利用其他 HRQOL 工具和医生确定的血管炎测量方法，确定 AAV 中 PROMIS 工具的结构有效性疾病活动和损害作为结构。具体目标 2：通过将随时间的变化与临床评估、疲劳 SF-36 和患者对变化测量的总体印象进行比较，确定 PROMIS 仪器在 AAV 群体中的纵向有效性、响应性和临床重要差异。 具体目标 3：确定 PROMIS 评估在定义疾病方面超出 HRQOL 和 AAV 疾病活动标准测量的附加/补充价值。 具体目标 4：在 AAV 随机治疗临床试验中使用选定的 PROMIS 仪器。 具体目标 5：通过评估 i) 患者对仪器领域重要性的评级，建立对 AAV 临床研究最有用的 PROMIS 症状测量“工具箱”； ii) 描述疾病过程范围的有用性； iii) 在 RCT 中使用各种 PROMIS 工具的相对效率和可行性；然后为研究人员提供知情指导，将 PROMIS 整合到 AAV 的临床研究中。该研究将由血管炎临床研究联盟 (VCRC) 进行，该联盟是北美最大的合作临床研究基础设施，并利用现有的研究队列和资源，充分响应 NIH RFA for PROMIS，包括 i) VCRC Longitudinal AAV 研究队列； ii) AAV 的 RITAZAREM 临床试验； iii) 在线 VCRC 患者联系登记处。 公共健康相关性：拟议的项目将探索新方法，将血管炎患者对其疾病负担的感受纳入临床研究，以便更好地了解这种疾病，并帮助开发一套研究工具来衡量这种疾​​病患者的观点。该项目将测试患者报告结果测量系统（PROMIS），这是一个由 NIH 开发的高度先进的系统，用于测量患者对疾病相关健康问题的评分。该研究将取得重要进展，提供一种将患者自己对疾病的评估纳入研究的方法。该项目与国家卫生研究院的使命和目标完全相关。