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

• 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
• 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

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.

项目总结/摘要 候选人：Patricia Vega-Fernandez，医学博士，理学硕士，RhMSUS是一名儿科风湿病学家， 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预后， 作为一名独立的临床科学家，专注于基于生物学的成像和临床研究，以支持 发展个性化的治疗方法。维加-费尔南德斯博士，儿科助理教授 在辛辛那提儿童医院医疗中心（CCHMC），进行了多项研究，重点是 探讨肌肉骨骼超声（MSUS）在幼年特发性关节炎中的可行性和可靠性 （JIA）.拟议的K23职业发展计划将重点关注维加-费尔南德斯博士 知识和培训是必不可少的，以推动她的职业生涯成为一个独立的调查员：1）关键 评价JIA的生物学特征：2）超声引导下滑膜活检在JIA患儿中的应用; 3)仪器和纵向研究的验证; 4）加强个人，学术和专业技能 成为一名独立资助的临床科学家。 导师/环境：Vega-Fernandez博士及其导师，Hermine Brunner MD MSc MBA，Sherry Thornton博士，Jonathan Dillman MD MSc和Jareen Meinzen-Derr PhD MPH组建了一支强大的团队 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：JIA是儿童时期最常见的慢性风湿性疾病，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活动范围受限的存在 和/或触诊有压痛。考虑到儿童在诊断时的年龄很小， 运动的精确性往往是不准确的。此外，临床评估活动性关节炎的发展， 骨架是主观的，并且具有低的评分者间可靠性。MSUS可以客观地告知存在和严重程度， 关节炎关于儿童MSUS结果的临床意义存在知识差距。中央 本研究的假设是，10关节聚焦MSUS评分可以作为诊断成像工具， 准确评估JIA患儿随时间推移的临床和生物学炎症活动。指导 通过强有力的初步数据，这一假设将被测试与三个具体目标：1）确定收敛 MSUS-10评分在JIA疾病活动范围内的有效性; 2）确定MSUS-10评分的能力。 10分，以捕获JIA的临床相关变化; 3）评价MSUS-10评分与 JIA炎症的生物标志物。建议的实验是有意义的，因为MSUS-10 检查和评分可以支持更安全和更适当的选择，用于治疗关节炎， 转将改善儿童与JIA的结果。这个项目是创新的，因为这种新颖的方法， MSUS评分可能导致JIA患者接受的治疗和护理的范式转变。