Mentoring and Patient Oriented Research in Juvenile Spondyloarthritis

青少年脊柱关节炎的指导和以患者为导向的研究

• 批准号: 10370698
• 负责人: Pamela Fitch Weiss
• 金额: $ 13.27万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-08-01 至 2027-07-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10370698
• 关键词: Accounting; Address; Adolescent; Adult; Advisory Committees; Anatomy; Ankylosing spondylitis; Arthritis; Bone Marrow; Caring; Child; Child Care; Childhood; Chronic Childhood Arthritis; Classification; Clinical; Clinical Research; Clinical Trials; Data; Data Sources; Diagnostic Imaging; Diffusion; Diffusion Magnetic Resonance Imaging; Disease; Disease remission; Early Diagnosis; Edema; Ensure; Environment; Faculty; Funding; Goals; Image; Image Analysis; Imaging Techniques; Inflammation; Inflammatory; Interleukin-17; International; Joints; Lesion; Location; Magnetic Resonance Imaging; Mentors; Methodology; NIH Program Announcements; Outcome; Pathologic; Pathology; Pathway interactions; Pelvis; Physicians; Population; Predictive Value; Public Health; Recommendation; Research; Research Methodology; Research Personnel; Rheumatology; Risk; Sacroiliac joint structure; Scientist; Societies; Specificity; Spondylarthritis; Suggestion; Techniques; Testing; Time; Training; Work; base; career development; clinical care; design; diagnostic accuracy; diagnostic biomarker; effectiveness study; efficacy study; epidemiology study; experience; functional disability; imaging biomarker; imaging study; improved; indexing; inhibitor; innovation; interleukin-23; next generation; novel; patient oriented research; peer; prevent; programs; quantitative imaging; rheumatologist; skills; subchondral bone; success; targeted treatment; tertiary care; tool

PROJECT SUMMARY Dr. Pamela Weiss, with the support of this proposal, seeks to expand and improve her research program and her training, mentoring, and career development of greatly needed physician scientists pursuing patient- oriented research (POR) in pediatric rheumatology. Successful mentoring requires both an effective mentee and a skilled mentor, both of which are reflected in the training goals of this application and include building skills to 1) train junior faculty and peers to build and effectively manage their mentoring relationships, and 2) teach trainees how to effectively work with their mentoring team and navigate the research environment. The premise of this application is to leverage existing studies to improve the everyday clinical care of children with spondyloarthritis and to contribute to the design of studies of emerging targeted therapies. Three major obstacles block advancements in juvenile spondyloarthritis care. First, there is variability in the interpretation of diagnostic imaging studies because distinguishing immature structural bone from subchondral bone marrow edema at the sacroiliac joints in children is challenging. The apparent diffusion coefficient (ADC) from diffusion- weighted imaging (DWI) holds unique promise for the pediatric population as a tool to distinguish maturational changes from pathologic inflammation. Second, there are no pediatric diagnostic imaging criteria for sacroiliitis. Improved criteria is needed not only in the clinical setting to prevent overdiagnosis, but also in the research setting to accurately identify those who would benefit from clinical trials. Third, although there is a juvenile spondyloarthritis disease activity (JSpADA) index that is widely used in clinical research, the cut-off values for defining clinically important disease states are unknown. Since inactive disease remains unattainable for many with juvenile spondyloarthritis, there is a critical need to determine minimal clinically important improvement (MCII) and well-defined states of disease activity using the JSpADA index for use not only in clinical care but also studies of emerging therapies. The proposed research addresses these challenges and leverages existing studies conducted by the candidate at a world-renowned pediatric tertiary care center with expertise in spondyloarthritis and innovative imaging. Aim 1 will test ADC from DWI as a diagnostic biomarker for the presence of sacroiliitis in children. Aim 2 will determine candidate lesion-based criteria for defining a positive sacroiliac joint MRI. Aim 3 will determine the MCII and cut-off values for defining minimal disease activity and high disease activity for the JSpADA index. These aims will simultaneously provide significant added value to ongoing studies, aid the applicant to develop new research and mentoring skills, and offer a clinical research platform in which to mentor young physician scientists in POR and promote their transition to independence.

项目概要 帕梅拉·韦斯 (Pamela Weiss) 博士在该提案的支持下，寻求扩大和改进她的研究计划， 她对急需的医师科学家进行培训、指导和职业发展，以追求患者 儿科风湿病学定向研究（POR）。成功的指导需要有效的受指导者 和熟练的导师，两者都反映在该应用程序的培训目标中，包括构建 1）培训初级教师和同行建立并有效管理他们的指导关系的技能，以及2） 教导学员如何有效地与他们的指导团队合作并驾驭研究环境。这 该应用程序的前提是利用现有研究来改善儿童的日常临床护理 脊柱关节炎并为新兴靶向疗法的研究设计做出贡献。三大 阻碍青少年脊柱关节炎护理进步的障碍。首先，解释上存在差异 诊断成像研究，因为可以区分未成熟的结构骨和软骨下骨髓 儿童骶髂关节水肿具有挑战性。表观扩散系数 (ADC) 来自扩散- 加权成像 (DWI) 作为区分成熟期的工具，对儿科人群具有独特的前景。 病理性炎症的变化。其次，尚无骶髂关节炎的儿科影像诊断标准。 不仅在临床环境中需要改进标准以防止过度诊断，而且在研究中也需要改进标准 设置以准确识别那些将从临床试验中受益的人。三、虽有少年 临床研究中广泛使用的脊柱关节炎疾病活动度（JSpADA）指数，其临界值 临床上重要的疾病状态的定义尚不清楚。由于非活动性疾病对许多人来说仍然是遥不可及的 对于幼年型脊柱关节炎，迫切需要确定最小的临床重要改善 (MCII) 和使用 JspADA 指数明确定义的疾病活动状态，不仅用于临床护理，还用于 还研究新兴疗法。拟议的研究解决了这些挑战并利用了现有的 由候选人在世界著名的儿科三级护理中心进行的研究，该中心拥有以下方面的专业知识 脊柱关节炎和创新成像。目标 1 将测试 DWI 中的 ADC 作为诊断生物标志物 儿童存在骶髂关节炎。目标 2 将确定基于候选病变的标准来定义阳性 骶髂关节 MRI。目标 3 将确定 MCII 和临界值，以定义最小疾病活动度和 JspADA 指数的疾病活动度较高。这些目标将同时提供显着的附加值 正在进行的研究，帮助申请人发展新的研究和指导技能，并提供临床研究 指导年轻医师科学家 POR 并促进他们向独立过渡的平台。