Enhancing Clinical Meaningfulness And Usefulness Of PROMIS Pediatric Measures Via Validation In Children And Adolescents With Rheumatic Disease, Cancer, Or Inflammatory Bowel Disease

 描述(由申请人提供)：已经开发了PROMIS(R)措施，以提供一种系统的、基于领域的方法来评估患者报告的结果(PRO)跨寿命和疾病组；然而，这些工具仍然没有被研究界，包括儿科疾病研究人员充分利用。这项PEPR建议利用儿童关节炎和风湿病研究联盟(CARA)、癌症儿科PRO研究网络以及美国克罗恩和结肠炎基金会合作伙伴儿童和青少年互联网队列这三个现有基础设施，进一步研究遗留的和新开发的PROIS措施，目的是加强和促进儿科研究人员和临床医生对这些措施的使用。仍然迫切需要采取积极措施，与疾病活动并行，识别高危患者，并反映对患者和家庭重要的健康结果。此外，更深入地了解患者因素与疾病负担之间的关系将促进成功的护理方法，并改善身心健康和社会福祉。我们对大约1500名患有风湿病、癌症和炎症性肠病的儿童进行了多中心纵向研究，提出了两个研究项目。第一个研究项目解决了PROMIS儿科措施临床验证的必要性，包括以下3个目标：目的1.评估PROMIS儿科措施的响应性，以衡量随着时间的推移健康变化及其与临床锚和患者报告的症状毒性的关系。目的2.评估抑郁和焦虑的PROMIS测量是否与随后的健康状况测量相关，包括身体和社会功能、物质使用和疾病特定结果；目的3.确定所采取的步骤(计步器数据)和PROMIS儿科测量之间的关联，以便探索在研究和临床护理中使用计步法数据来增加PRO。第二个研究项目解决了提高PROMIS测量的意义和有用性的需要，并包括以下3个目标：目标4.使用潜伏期分析和潜伏期分析来确定患有风湿病、癌症或IBD的儿童在身心健康方面未被观察到的亚群，并检查与亚组分类和随时间转移相关的人口学和临床特征；目标5.从儿童的角度评估PROMIS评分变化的临床最小重要差异(MID)，并与先前通过定性量表-判断方法产生的MID进行比较；目的6.使用青少年、父母和临床医生的标准设置方法，确定与不同程度的症状严重程度和功能状态相关的PROMIS儿科指标的临床切割评分。该中心的数据管理和管理核心包括经验丰富的临床医生、行为科学家、统计学家、心理测量学家和信息技术专家，以促进、协调和协同这些研究研究。

 DESCRIPTION (provided by applicant): PROMIS(r) measures have been developed to provide a systematic, domain-based approach to the assessment of patient-reported outcomes (PROs) across the lifespan and disease groups; however, these instruments continue to be underutilized by the research community, including pediatric disease investigators. This PEPR proposal leverages three existing infrastructures of the Childhood Arthritis and Rheumatology Research Alliance (CARRA), Cancer Pediatric PRO Research Network, and the Crohn's and Colitis Foundation of America Partners Kids and Teens Internet Cohort to further study legacy and newly developed PROMIS measures with the goal to enhance and promote their use by pediatric researchers and clinicians. There continues to be compelling need for PRO measures that parallel disease activity, identify high-risk patients, and reflect health outcomes important o patients and families. In addition, more robust understanding of relationships between patient factors and disease burden will promote successful care approaches and improved physical and mental health and social well-being. Two research projects are proposed for our multicenter longitudinal study of approximately 1500 children with rheumatic disease, cancer, and inflammatory bowel disease. The first research project addressed the need for clinical validation of PROMIS Pediatric measures including the following 3 aims: Aim 1. Evaluate the responsiveness of the PROMIS Pediatric measures to measure change in health over time and its association with clinical anchors and patient-reported symptom toxicities. Aim 2. Evaluate whether PROMIS measures of depression and anxiety are associated with subsequent measures of health status including physical and social functioning, substance use, and disease specific outcomes; Aim 3. Determine the association between steps taken (pedometer data) and PROMIS Pediatric measures in order to explore the use of pedometry data to augment PROs in research and clinical care. The second research project addresses the need to enhance the meaningfulness and usefulness of PROMIS measures and includes the following 3 aims: Aim 4. Identify unobserved subgroups of children with rheumatic disease, cancer, or IBD with respect to physical and mental health using latent profile analysis and latent transition analysis, and examine demographic and clinical characteristics associated with the subgroup classification and transition over time; Aim 5. Estimate a clinically minimally important difference (MID) in change in PROMIS Pediatric scores from the child's perspective and compare with the previously generated estimate of an MID of 3 points from qualitative scale-judgment methods; and Aim 6. Identify clinical cut scores along the PROMIS Pediatric metric associated with varying levels of symptom severity and functional status using standard setting methods with adolescents, parents, and clinicians. The Center's Data Management and Administrative Cores include experienced clinicians, behavioral scientists, statisticians, psychometricians, and information technology experts to facilitate, coordinate, and synergize these research studies.