Outcomes Research in Rheumatic Diseases

K24申请的目标是支持我作为导师的持续发展和影响，在密歇根大学（UM）的风湿病学部门建立一个面向患者的研究（POR）的研究培训计划，并扩展我目前的POR计划。在过去的几年里，我积极指导不同POR项目的学员和初级教员，拟议的K24的直接目标是确保保护时间，继续我的努力，并培养未来的临床研究者，重点是患者报告的结果测量和风湿病的试验设计。除了指导一个大型硬皮病项目外，我继续广泛研究其他关节炎（痛风和类风湿性关节炎），并得到系统性红斑狼疮和纤维肌痛的成熟临床研究人员的支持，为UM提供了丰富的POR环境。鉴于我对指导的承诺，以及最近与澳大生物统计学系的合作，澳大丰富的POR资源，以及创建这个项目的强大机构支持，K24奖提供了一个宝贵的机会，可以扩大我的指导工作，并建立一个专门的结构化POR项目。该计划将确保有兴趣成为独立研究者的医学和生物统计学学生和研究员将得到跨学科导师团队的支持，并将获得临床研究方法和生物统计学方面的培训。K24基金将节省我50%的工作时间，减轻我未来的临床和行政责任。目前资助的研究机会的多样化组合存在于贫穷和贫困地区的未来学员。拟议的研究包括两个当前项目和两个新项目，涉及患者报告结果、临床试验设计和数据库开发的贫困领域。目前NIH资助的两项研究包括：1。开发和评估美国国立卫生研究院患者报告结果测量信息系统倡议下开发的胃肠道症状题库的心理测量特性；和2。在一项正在进行的NIH资助的纵向观察研究中，为硬皮病临床试验制定一个联合反应指数。两项新提出的研究的目的是：在一项正在进行的NIH资助的临床试验中，比较150例活动性SSc-ILD患者口服霉酚酸酯与口服环磷酰胺，以确定维生素D状态与硬皮病相关间质性肺病（SSc-ILD）的生理、放射学和呼吸困难指标之间的关系。2. 通过美国大型硬皮病中心的几位年轻研究人员的协调努力，建立早期弥漫性硬皮病观察登记。登记的目标包括：i)确定早期弥漫性硬皮病的临床表型和自身抗体基线是否与临床、放射学和患者报告的结果和生存相关，并预测这些结果和生存；ii)开发一个存储库

DESCRIPTION (provided by applicant): Outcomes research in rheumatic diseases The goals of the K24 application are to support my continued development and impact as a mentor, to establish a research training program for patient-oriented research (POR) in the Division of Rheumatology at the University of Michigan (UM), and to expand my current POR program. In the past few years, I have actively mentored trainees and junior faculty on different POR projects and the immediate objective of the proposed K24 is to secure protected time to continue my efforts and develop future clinical investigators with a focus on patient-reported outcome measures and trial design in rheumatic diseases. In addition to directing a large Scleroderma program, I continue to work extensively in other arthritides (gout and rheumatoid arthritis) and have support of established clinical researchers in systemic lupus erythematosus and fibromyalgia, providing a rich environment of POR at UM. Given my commitment to mentoring, recent collaborations with the UM Department of Biostatistics, UM's rich resources dedicated to POR, and strong institutional support to create this program, the K24 Award provides an invaluable opportunity to expand my mentoring efforts and to establish a specialized structured POR program. The program will ensure that medical and biostatistical students and fellows interested in becoming independent investigators will be supported by an interdisciplinary team of mentors and will acquire training in clinical research methods and biostatistics. K24 grant would preserve my time at 50% effort and relieve future clinical and administrative responsibilities. A diverse portfolio of currently funded research opportunities exists for future trainees in POR. Research proposed includes two current projects and two new projects addressing POR areas of patient reported outcomes, clinical trial design, and database development. The two current NIH funded studies include: 1. To develop and evaluate the psychometric properties of gastrointestinal symptoms item bank developed under the NIH Patient-Reported Outcomes Measurement Information System initiative; and 2. To develop a combined response index for scleroderma clinical trials in an ongoing NIH funded longitudinal observational study. The aims of two new proposed studies are: 1. To determine the relationship between the vitamin D status and physiological, radiological, and dyspnea measures of scleroderma-associated interstitial lung disease (SSc-ILD) in an ongoing NIH funded clinical trial comparing oral mycophenolate mofetil vs. oral cyclophosphamide in 150 patients with active SSc-ILD. 2. To develop an early diffuse scleroderma observational registry through the coordinated efforts of several young investigators at large U.S. scleroderma centers. The goals of the registry include: i) to determine whether the clinical phenotypes and autoantibodies at baseline in early, diffuse scleroderma are associated with, and predict, clinical, radiological, and patient-reported outcomes and survival; and ii) to develop a repository of scleroderma blood and tissue to explore the cellular and molecular mediators of SSc and their association with clinical phenotypes and autoantibodies.