Adaption and Validation of PROMIS for use in Vasculitis

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ANCA相关血管炎患者的疾病活动性和负担的评估中[AAV、肉芽肿病伴多血管炎(Wegener‘s)、显微镜下的多血管炎和Churg-Strauss综合征]。本项目旨在证明将患者报告结果测量系统(PROIS)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：通过对脉管炎临床研究联盟(VCRC)患者队列中的受试者进行横断面分析，利用其他HRQOL工具和医生确定的脉管炎疾病活动和损害的测量作为结构，确定PROMIS工具在AAV中的结构有效性。具体目标2：通过比较随时间的变化与临床评估、疲劳SF-36和患者对变化的总体印象的测量，确定PROMIS工具在AAV人群中的纵向有效性、响应性和临床重要性差异。具体目标3：确定PROMIS评估在确定疾病时的相加/补充价值，超出AAV的HRQOL和疾病活动性的标准衡量标准。具体目标4：在AAV的随机治疗临床试验中使用精选的PROMIS器械。具体目标5：通过评估i)患者对仪器领域重要性的评级；ii)描述疾病过程的程度的有用性；iii)在随机对照试验中使用各种PROMIS仪器的相对效率和可行性；然后为研究人员提供知情指导，以将PROMIS纳入AAV的临床研究，从而建立一个对AAV临床研究最有用的PROMIS症状测量“工具箱”。这项研究将由北美最大的协作性临床研究基础设施脉管炎临床研究联盟(VCRC)进行，并通过利用已经建立的研究队列和资源，充分响应NIH PROMIS的RFA，包括i)AAV的VCRC纵向研究队列；ii)AAV的RITAZAREM临床试验；以及iii)在线VCRC患者联系登记。

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.