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.

项目摘要帕梅拉·魏斯（Pamela Weiss）博士在这项提案的支持下，试图扩大和改善她的研究计划和她的培训，指导和职业发展非常需要的医师科学家追求患者 - 小儿风湿病学的定向研究（POR）。成功的指导需要有效的受训者还有一个熟练的导师，这两者都反映在本应用程序的培训目标中，包括建造 1）培训初级教师和同龄人建立和有效管理他们的指导关系的技能，2）教学员如何与他们的指导团队有效合作并浏览研究环境。这此应用的前提是利用现有研究来改善儿童的日常临床护理脊椎关节炎，并为新兴靶向疗法的研究做出贡献。三个主要障碍阻碍了少年脊柱肝炎护理的进步。首先，解释的可变性诊断成像研究是因为将未成熟的结构骨与软骨下骨髓区分开儿童的sa骨关节的水肿具有挑战性。从扩散 - 明显的扩散系数（ADC）加权成像（DWI）对小儿种群具有独特的希望，作为区分成熟的工具病理炎症的变化。其次，没有儿科诊断成像标准s骨炎。不仅在临床环境中需要改进标准以防止过度诊断，而且还需要设置可以准确识别那些将从临床试验中受益的人。第三，尽管有少年在临床研究中广泛使用的脊椎关节炎疾病活性（JSPADA）指数，定义临床上重要的疾病状态尚不清楚。由于无活性疾病仍然无法实现少年脊柱糖关节炎，确定最小临床重要的改善至关重要（MCII）使用JSPADA指数不仅在临床护理中使用，而且可以使用明确定义的疾病活动状态还研究新兴疗法。拟议的研究解决了现有的这些挑战和利用候选人在世界知名的儿科三级护理中心进行的研究具有专业知识脊椎关节炎和创新成像。 AIM 1将测试DWI的ADC作为诊断生物标志物儿童存在s骨炎。 AIM 2将确定基于候选病变的标准，以定义阳性 sacroiliac关节MRI。 AIM 3将确定MCII和截止值，以定义最小疾病活动和 JSPADA指数的高疾病活动。这些目标将同时为正在进行的研究，帮助申请人发展新的研究和指导技能，并提供临床研究在POR中指导年轻医师科学家并促进其向独立过渡的平台。