Adaption and Validation of PROMIS for use in Vasculitis

PROMIS 用于血管炎的调整和验证

• 批准号: 8545674
• 负责人: Peter A Merkel
• 金额: $ 38.14万
• 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/8545674
• 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.

描述（由申请方提供）：拟议工作的总体目标是更有效地将患者的观点纳入ANCA相关血管炎患者[AAV、肉芽肿伴多血管炎（韦格纳）、显微镜下多血管炎和Churg-Strauss综合征]的疾病活动和负担评估中。本项目旨在证明将患者报告结局测量系统（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疾病活动和损伤指标作为结构，确定PROMIS工具在AAV中的结构有效性。具体目标二：通过将随时间推移的变化与临床评价、疲劳SF-36和患者总体印象变化指标进行比较，确定PROMIS工具在AAV人群中的纵向有效性、响应性和临床重要差异。 具体目标3：确定PROMIS评估在定义AAV中HRQOL和疾病活动性标准测量之外的疾病方面的附加/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。 具体目标五：建立PROMIS症状测量的“工具箱”，通过评估i）患者对工具域重要性的评级; ii）描述疾病过程程度的有用性;以及iii）在RCT中使用各种PROMIS工具的相对效率和可行性，该工具箱对AAV的临床研究最有用;然后为研究人员提供知情的指导，将PROMIS整合到AAV的临床研究中。本研究将由血管炎临床研究联盟（VCRC）进行，VCRC是北美最大的合作临床研究基础设施，通过利用已建立的研究队列和资源，包括i）AAV的VCRC纵向研究队列; ii）AAV的RITAZAREM临床试验;和iii）在线VCRC患者接触登记，完全响应了NIH RFA的PROMIS。