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

项目摘要/摘要 候选人：Patricia Vega-Fernandez，医学硕士，RhMSUS是一位儿科风湿病专家，其主要职业生涯 目标是通过职业生涯改善受自身免疫性风湿病影响的儿童的结局 作为一名专注于基于生物的成像和临床研究的独立临床科学家， 开发个性化治疗方法。儿科学助理教授维加-费尔南德斯医生 在辛辛那提儿童医院医学中心(CCHMC)，进行了多项研究，重点是 探讨肌肉骨骼超声在青少年特发性关节炎诊断中的可行性和可靠性 (贾)。拟议的K23职业发展计划将重点放在维加-费尔南德斯博士 将她的职业生涯推向独立调查员所必需的知识和培训：1)关键 评价JIA的生物学特征：2)超声引导下对JIA患儿进行滑膜活检； 3)验证工具和纵向研究；4)加强个人、学术和专业技能 成为一名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医学硕士MBA Sherry 桑顿博士、乔纳森·迪尔曼医学硕士和Jareen Meinzen-Derr博士公共卫生硕士组成了一个强大的团队 来自CCHMC内外的顾问和合作者，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：贾是儿童最常见的慢性风湿病，以关节炎症为特征 (关节炎)。儿童关节炎的诊断是基于关节肿胀和有限的活动范围。 和/或触诊时的触痛。鉴于确诊时儿童的年龄较小，关节疼痛或受限的报告 通常情况下，运动的精度可能不准确。此外，活动期关节炎的临床评估仍在发展中 主观性较强，评分者间信度较低。MSU可以客观地告知存在和严重程度 关节发炎。对于MSUS在儿童中发现的临床意义，人们存在着认识上的差距。中环 这项研究的假设是，10个关节的MSUS评分可以作为一种诊断成像工具，以提供 准确评估JIA儿童随时间推移的临床和生物学炎症活动。引导式 通过强大的初步数据，这一假设将以三个具体的目标进行检验：1)确定收敛 MSU-10评分在JIA疾病活动范围内的有效性；2)确定MSU-10的能力- 10分来捕捉JIA的临床相关变化；3)评估MSUS-10评分与 JIA炎症的生物学标志物。拟议的实验意义重大，因为MSU-10 检查和评分可能支持更安全和更合适的关节炎治疗选择，在 TURN将改善JIA患儿的预后。这个项目是创新的，因为这种新的方法使用 MSUS评分可能导致JIA患者接受的治疗和护理的范式转变。

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.