Validation of a Novel Ultrasound Score to Improve the Assessment of Joint Inflammation in Children with Juvenile Arthritis

验证新型超声评分以改善幼年关节炎儿童关节炎症的评估

基本信息

批准号：
10739429
负责人：
Patricia Vega Fernandez
金额：
$ 17.01万
依托单位：
CINCINNATI CHILDRENS HOSP MED CTR
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-08-01 至 2028-07-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10739429
关键词：
Address Affect Age Alpha Interleukin 2 Receptor Angiopoietin-2 Angiopoietins Arthralgia Arthritis Articular Range of Motion Award Biological Biological Assay Biological Markers Biology Biopsy Career Mobility Caring Child Childhood Chronic Chronic Childhood Arthritis Classification Clinical Clinical Markers Clinical Research Clinical assessments Data Detection Development Development Plans Diagnosis Diagnostic Imaging Disease Doctor of Philosophy Enrollment Environment Evaluation Foundations Functional disorder Funding Goals Healthcare Systems Histologic Image Imaging Device Immunology Inflammation Inflammatory Interferon Type II Interleukin 2 Receptor Interleukin-6 Joint Laxity Joints Knowledge Longitudinal Studies Measurement Measures Medical center Mentors Mentorship Motion Outcome Pain Palpation Patients Pediatric Hospitals Pediatrics Performance Pharmaceutical Preparations Physical Examination Pilot Projects Plasma Protocols documentation Provider Publishing Quality of life Reference Standards Reporting Research Research Activity Research Personnel Resources Rheumatism Rheumatoid Arthritis Rheumatology Sampling Scientist Serum Severities Skeleton Standardization Swelling Symptoms Synovial Fluid Synovitis System TNF gene Testing Time Tissues Training Training Activity Ultrasonography United States National Institutes of Health Universities Validation Vascular Endothelial Growth Factors Visit adverse event risk aged autoimmune rheumatologic disease candidate marker career career development clinically relevant clinically significant experience experimental study follow-up functional disability imaging biomarker improved improved outcome innovation instrument joint inflammation joint injury musculoskeletal ultrasound novel novel strategies personalized medicine point of care professor response rheumatologist skills treatment choice treatment response ultrasound

项目摘要

PROJECT SUMMARY/ABSTRACT Candidate: Patricia Vega-Fernandez, MD MSc RhMSUS is a pediatric rheumatologist whose overarching career goal is to improve the outcomes of children affected by autoimmune rheumatologic diseases through a career as an independent clinician scientist focused on biology-based imaging and clinical research to support the development of personalized treatment approaches. Dr. Vega-Fernandez, an Assistant Professor of Pediatrics at Cincinnati Children’s Hospital Medical Center (CCHMC), has conducted multiple studies focused on addressing the feasibility and reliability of musculoskeletal ultrasound (MSUS) in Juvenile Idiopathic Arthritis (JIA). The proposed K23 career development plan will focus on identified gaps in Dr. Vega-Fernandez’s knowledge and training that are essential to advance her career towards an independent investigator: 1) Critical appraisal of the biological signatures in JIA; 2) Performance of US-guided synovial biopsy in children with JIA; 3) Validation of instruments and longitudinal studies; 4) Strengthened personal, academic, and professional skills to become an effective independently funded clinical-scientist. Mentors/Environment: Dr. Vega-Fernandez and her mentors, Hermine Brunner MD MSc MBA, Sherry Thornton PhD, Jonathan Dillman MD MSc, and Jareen Meinzen-Derr PhD MPH, have assembled a strong team of advisors and collaborators from inside and outside of CCHMC to guide the proposed training and research activities. The career development plan utilizes intellectual resources at CCHMC and the University of Cincinnati. Research: JIA, the most common chronic rheumatic disease in childhood, is characterized by joint inflammation (arthritis). Diagnosis of arthritis in children is based on the presence of joint swelling and limited range of motion and/or tenderness on palpation. Given the young age of children at diagnosis, the report of joint pain or limitation of motion can often times be inaccurate. In addition, clinical assessment of active arthritis in the developing skeleton is subjective and has low inter-rater reliability. MSUS can objectively inform presence and severity of joint inflammation. There is a knowledge gap on the clinical significance of MSUS findings in children. The central hypothesis of this study is that a 10-joint focused MSUS score can serve as a diagnostic imaging tool to provide an accurate assessment of both clinical and biologic activity of inflammation over time in children with JIA. Guided by strong preliminary data, this hypothesis will be tested with three specific aims: 1) Determine the convergent validity of the MSUS-10 score across the spectrum of JIA disease activity; 2) Determine the ability of the MSUS- 10 score to capture clinically relevant change of JIA; 3) Evaluate the relationship of the MSUS-10 score with biologic markers of JIA inflammation. The proposed experiments are significant because the MSUS-10 examination and score may support safer and more appropriate choices for the treatment of arthritis, which in turn will improve the outcomes of children with JIA. This project is innovative as this novel approach of using MSUS scoring may lead to a paradigm shift in the treatment and care that JIA patients receive.

项目摘要/摘要候选人：帕特里夏·维加·费尔南德斯（Patricia Vega-Fernandez）目标是改善在职业生涯中受到自身免疫性风湿病影响的儿童的结果作为一名独立临床科学家，专注于基于生物学的成像和临床研究，以支持开发个性化治疗方法。 Vega-Fernandez博士，儿科助理教授在辛辛那提儿童医院医疗中心（CCHMC），已进行了多项研究解决少年特发性关节炎中肌肉骨骼超声（MSU）的可行性和可靠性（贾）。拟议的K23职业发展计划将重点介绍Vega-Fernandez博士的差距知识和培训对于将职业发展到独立调查员至关重要：1）关键评估JIA的生物学特征； 2）在JIA儿童中，US引导的滑膜活检表现； 3）验证工具和纵向研究； 4）增强个人，学术和专业技能成为有效的独立资助的临床科学家。导师/环境：Vega-Fernandez博士和她的导师Hermine Brunner MD MSC MBA，雪利酒 Thornton PhD，Jonathan Dillman MD MSC和Jareen Meinzen-Derr PhD MPH，已经组建了一支强大的团队来自CCHMC内部和外部的顾问和合作者，以指导拟议的培训和研究活动。职业发展计划利用CCHMC和辛辛那提大学的智力资源。研究：贾（Jia）是儿童期最常见的慢性风湿病，其特征是关节炎症（关节炎）。儿童关节炎的诊断是基于关节肿胀和运动范围有限的和/或触诊时温度。鉴于儿童诊断时的年龄很小，关节疼痛或限制的报告运动通常可能不准确。此外，发育中的主动关节炎的临床评估骨骼是主观的，评估者间的可靠性较低。 MSU可以客观地告知其存在和严重性关节炎症。关于儿童MSU的发现的临床意义存在知识差距。中央这项研究的假设是，以10关节为主的MSU分数可以用作诊断成像工具对JIA儿童的炎症随时间的临床和生物学活性的准确评估。指导通过强大的初步数据，该假设将通过三个特定目的进行检验：1）确定转换 MSUS-10评分在整个JIA病活性范围内的有效性； 2）确定msus-的能力 10分，以捕获JIA的临床相关变化； 3）评估MSUS-10分数与 JIA注射的生物标记。提出的实验很重要，因为MSUS-10 检查和评分可能支持治疗关节炎的更安全，更合适的选择，这在转弯将改善JIA儿童的结果。这个项目具有创新性，因为这种新颖的方法 MSUS评分可能会导致JIA患者接受的治疗和护理的范式转移。