Outcomes of children with juvenile idiopathic arthritis-associated uveitis

首席调查员/项目主任(最后、第一、中间)：洛杉矶-韩，希拉，T 项目摘要 幼年特发性关节炎相关性葡萄膜炎(JIA-U)是一种炎症性眼病，影响20%的 儿童患有JIA，可导致白内障、青光眼、失明和失明。现行筛查指南 旨在识别JIA中有眼病风险的儿童。然而，儿科风湿科医生和 眼科医生为葡萄膜炎提供全面筛查的能力是有限的，因为指南 没有更新到包括所有青少年关节炎亚型和其他已知的视力限制风险因素 JIA的并发症。对JIA和其他相关的儿童自身免疫性疾病进行筛查是至关重要的，因为 早期发现葡萄膜炎可预防视力并发症，影响治疗效果。同样， 必要时早期积极治疗可能会避免严重的视力并发症。增进健康 对于患有JIA-U的儿童的预后和生活质量，我们需要更好地了解葡萄膜炎的危险因素， 长期视力结果，以及视力和身体残疾对儿童生活的影响。早早地和 通过确定葡萄膜炎的易感性和严重程度的危险因素准确地鉴定JIA-U， 结果衡量标准的标准化和人类白细胞抗原风险等位基因重要性的阐明将改善 评估JIA-U的影响以及对疾病病因、发病机制和转归的了解。 同样，改进的风险分层计划可以减少不必要的眼科就诊，并帮助我们 找出患严重眼病和并发症风险较高的儿童。 本研究的目的是：1)明确JIA-U在临床特征和病程上的差异。 在患有JIA的儿童中识别临床和遗传风险标记的欧洲和非洲裔患者 葡萄膜炎，2)对一组确诊为葡萄膜炎的儿童进行为期4年的前瞻性研究，检查确定的危险因素 用早期的JIA来确定哪些因素与葡萄膜炎的最终发展有关，并提供数据 在它们的纵向轮廓上，3)确定关节炎和葡萄膜炎对身体和视觉的影响 使用主观和客观测量的残疾、特定视力的生活质量和总体生活质量。使用 综合生物、遗传和心理社会方法，我们希望开发出更广泛适用的风险 基于JIA亚型的分层方案，并确定新的风险因素，以确定 严重残疾和失明风险最大的儿童，以便制定干预措施，以改善 他们的总体结果。 这位候选人是一名儿科风湿学家，也是埃默里大学的儿科学助理教授。她 已获得临床调查理学硕士学位。她致力于临床成果方面的事业 对患有自身免疫性眼病的儿童的研究。她组建了一支出色的指导团队， 在儿科风湿病、儿科眼科、儿科流行病学、结果研究和 测量，这与她的目标相关。这个导师奖将帮助她成为一名成功的 独立的临床医生科学家。 项目说明第7页

Principal Investigator/Program Director (Last, first, middle): Angeles-Han, Sheila, T Project Summary Juvenile idiopathic arthritis-associated uveitis (JIA-U) is an inflammatory eye disease that affects 20% of children with JIA and can lead to cataracts, glaucoma, vision loss and blindness. Current screening guidelines are designed to identify children at risk for eye disease in JIA. However, pediatric rheumatologists' and ophthalmologists' ability to provide comprehensive screening for uveitis is limited because the guidelines have not been updated to include all juvenile arthritis subtypes and other known risk factors for vision-limiting complications of JIA. Screening in JIA and other related autoimmune diseases of childhood is crucial because early uveitis detection can prevent visual complications and influence therapeutic management. Likewise, institution of early aggressive therapy when needed may avert serious visual complications. To improve health outcomes and quality of life of children with JIA-U, we need a better understanding of risk factors for uveitis, long-term visual outcomes, and the effects of visual and physical disability on children's lives. Early and accurate identification of JIA-U through the identification of risk factors for susceptibility and severity of uveitis, standardization of outcome measures, and elucidation of the significance of HLA risk alleles would improve the evaluation of the impact of JIA-U and the understanding of disease etiology, pathogenesis and outcome. Likewise, an improved risk stratification scheme could reduce unnecessary ophthalmology visits and help us identify children at greater risk for severe eye disease and complications. The objectives of this study are to 1) Identify differences in the clinical characteristics and course of JIA-U in patients of European and African descent in children with JIA to identify clinical and genetic risk markers for uveitis, 2) Examine the risk factors identified over a 4-year prospective study of a cohort of children diagnosed with early JIA to determine which factors are associated with eventual development of uveitis, and provide data on their longitudinal profile, 3) Determine the impact of having both arthritis and uveitis on physical and visual disability, vision-specific QOL, and overall QOL using subjective and objective measures. Using a comprehensive biologic, genetic and psychosocial approach, we hope to develop more widely applicable risk stratification schemes based on JIA subtype, and to identify new risk factors that will identify the populations of children at greatest risk for severe disability and blindness so that interventions can be developed to improve their overall outcome. The candidate is a pediatric rheumatologist and an assistant professor of pediatrics at Emory University. She has obtained a Masters of Science in Clinical Investigation. She is devoted to a career in clinical outcomes research in children with autoimmune eye diseases. She has assembled an outstanding mentoring team with experience in pediatric rheumatology, pediatric ophthalmology, pediatric epidemiology, outcomes research and measurement, which are relevant to her objectives. This mentored award will help her become a successful independent clinician scientist. Project Description Page 7