Outcomes research in rheumatic diseases

描述(申请人提供)：风湿病研究成果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)风湿科建立以患者为导向的研究培训计划，并扩大我目前的POR计划。在过去的几年里，我在不同的POR项目上积极指导实习生和初级教师，拟议的K24的直接目标是确保有保护的时间来继续我的努力，并培养未来的临床研究人员，重点是患者报告的结果衡量标准和风湿病的试验设计。除了领导一个大型硬皮病项目外，我还继续在其他关节炎(痛风和类风湿性关节炎)方面广泛工作，并在系统性红斑狼疮和纤维肌痛方面得到知名临床研究人员的支持，为UM提供了丰富的POR环境。考虑到我对指导的承诺，最近与密歇根大学生物统计部门的合作，UM在POR方面的丰富资源，以及创建这个项目的强有力的机构支持，K24奖提供了一个宝贵的机会来扩大我的指导努力，并建立一个专门的结构化POR计划。该计划将确保有兴趣成为独立调查人员的医学和生物统计学学生和研究员将得到一个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 GRANT将节省我50%的时间，并减轻未来的临床和管理责任。目前为POR的未来受训人员提供了一系列多样化的研究机会。建议的研究包括两个当前项目和两个新项目，涉及POR领域的患者报告结果、临床试验设计和数据库开发。目前NIH资助的两项研究包括：1.开发和评估NIH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学特性；2.在正在进行的NIH资助的纵向观察性研究中，为硬皮病临床试验开发综合反应指数。两项新提出的研究的目的是：1.在NIH资助的一项正在进行的临床试验中，比较150名SSC-ILD患者口服霉酚酸酯和口服环磷酰胺，以确定维生素D状况与硬皮病相关性间质性肺疾病(SSC-ILD)的生理、放射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名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是否与临床、放射学和患者报告的结果和存活率有关，并可预测；ii)开发一个资料库 目的：探讨硬皮病血液和组织中SSC的细胞和分子介质及其与临床表型和自身抗体的关系。 公共卫生相关性K24应用程序的目标是支持我作为一名导师的持续发展，并为以患者为导向的研究建立研究培训计划。拟议的计划是跨学科的，重点是扩大风湿病的临床研究机会和指导。K24奖将确保我有保护的时间，能够扩大我目前的指导活动，同时继续推进我的风湿病患者以患者为中心的研究计划。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等以患者为中心的研究领域。

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. PUBLIC HEALTH RELEVANCE The goals of the K24 application are to support my continued development as a mentor and to establish a research training program for patient oriented research. The proposed program is interdisciplinary, and focuses in expanding clinical research opportunities and mentoring in rheumatic diseases. The K24 Award would ensure that I have the protected time to be able to expand my current mentoring activities while continuing to advance my patient oriented research program in patients with rheumatic disease. Research includes two current projects and two new projects addressing patient-oriented research areas of patient-reported outcomes, clinical trial design, and database development.