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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

基本信息

批准号：
10673614
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
项目类别：
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/10673614
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 Diagnosis Diagnostic Imaging Diffusion Diffusion Magnetic Resonance Imaging Disease Disease remission Early Diagnosis Edema Educational process of instructing Ensure Environment Faculty Funding Goals IL17 gene Image Image Analysis Imaging Techniques Inflammation Inflammatory International Joints Lesion Location Magnetic Resonance Imaging Mentors Methodology NIH Program Announcements Outcome Pathologic Pathology Pathway interactions Pediatric Radiologist Pelvis Physicians Population Predictive Value Public Health Recommendation Research Research Methodology Research Personnel Rheumatology Sacroiliac joint structure Scientist Societies Specificity Spondylarthritis Techniques Testing Time Training Work 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 progression risk quantitative imaging rheumatologist skills subchondral bone success targeted treatment tertiary care tool

项目摘要

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。