Outcomes Research in Rheumatic Diseases

风湿性疾病的结果研究

• 批准号: 8917860
• 负责人: DINESH KHANNA
• 金额: $ 17.96万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-09-01 至 2016-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8917860
• 关键词: 25-hydroxyvitamin D; Acute-Phase Proteins; Address; Area; Arthritis; Autoantibodies; Biometry; Blood; Blood capillaries; Characteristics; Chest; Clinical; Clinical Investigator; Clinical Research; Clinical Trials; Clinical Trials Design; Cognitive; Collaborations; Committee Members; Consensus; Cutaneous; Cyclophosphamide; Data; Databases; Development; Diffuse; Diffuse Scleroderma; Dyspnea; Ensure; Environment; Faculty; Fibromyalgia; Fibrosis; Focus Groups; Funding; Future; Gastrointestinal Diseases; Genetic Markers; Goals; Gout; Grant; High Resolution Computed Tomography; Inflammation; Information Systems; Interstitial Lung Diseases; Interview; Joints; Literature; Lung; Measurement; Measures; Mediator of activation protein; Medical; Mentors; Michigan; Mid-Career Clinical Scientist Award (K24); Modeling; Molecular; Mycophenolate; Nail Fold; National Heart, Lung, and Blood Institute; National Institute of Arthritis and Musculoskeletal and Skin Diseases; Observational Study; Oral; Outcome Measure; Outcomes Research; Patient Outcomes Assessments; Patients; Physiological; Placebos; Property; Psychometrics; Randomized Controlled Trials; Registries; Reporting; Research; Research Methodology; Research Personnel; Research Project Grants; Research Support; Research Training; Resources; Rheumatism; Rheumatoid Arthritis; Rheumatology; Role; Scleroderma; Secure; Serological; Serum; Skin; Structure; Students; Swelling; Systemic Lupus Erythematosus; Systemic Scleroderma; Taxonomy; Techniques; Time; Tissues; Training; Training Programs; United States National Institutes of Health; Universities; Validation; Vascular Diseases; Vitamin D; Work; X-Ray Computed Tomography; biobank; biopsychosocial; capillary; clinical phenotype; cohort; experience; gastrointestinal symptom; indexing; interest; mycophenolate mofetil; patient oriented research; programs; prospective; repository; response; systematic review; theories; trial comparing; trial design

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.

描述（由申请人提供）：风湿性疾病的成果研究 K24 申请的目标是支持我作为导师的持续发展和影响力，在密歇根大学 (UM) 风湿病科建立以患者为导向的研究 (POR) 的研究培训计划，并扩展我当前的 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师，拟议的 K24 的直接目标是确保有受保护的时间来继续我的努力并培养未来的临床研究人员，重点关注患者报告的结果测量和风湿性疾病的试验设计。除了指导大型硬皮病项目外，我还继续在其他关节炎（痛风和类风湿关节炎）方面进行广泛的工作，并得到系统性红斑狼疮和纤维肌痛领域成熟临床研究人员的支持，为密西根大学的 POR 提供了丰富的环境。鉴于我对指导的承诺、最近与 UM 生物统计系的合作、UM 致力于 POR 的丰富资源以及创建该计划的强大机构支持，K24 奖为我扩大指导工作并建立专门的结构化 POR 计划提供了宝贵的机会。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得到跨学科导师团队的支持，并获得临床研究方法和生物统计学方面的培训。 K24 资助将节省我 50% 的时间并减轻未来的临床和行政责任。目前资助的研究机会多种多样，可供 POR 未来的学员使用。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 POR 领域。目前 NIH 资助的两项研究包括： 1. 开发和评估根据 NIH 患者报告结果测量信息系统倡议开发的胃肠道症状项目库的心理测量特性； 2. 在 NIH 资助的一项正在进行的纵向观察研究中制定硬皮病临床试验的综合反应指数。两项新拟定研究的目的是： 1. 在 NIH 资助的一项正在进行的临床试验中，对 150 名活动性 SSc-ILD 患者进行比较口服吗替麦考酚酯与口服环磷酰胺的临床试验，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，开发早期弥漫性硬皮病观察登记册。该注册中心的目标包括： i) 确定早期弥漫性硬皮病基线时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生存相关并进行预测； ii) 开发一个存储库 硬皮病血液和组织，以探索 SSc 的细胞和分子介质及其与临床表型和自身抗体的关联。