Outcomes of children with juvenile idiopathic arthritis-associated uveitis

幼年特发性关节炎相关葡萄膜炎儿童的结局

• 批准号: 8165651
• 负责人: Sheila Therese Angeles-Han
• 金额: $ 19.6万
• 依托单位: EMORY UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-09-01 至 2016-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8165651
• 关键词: Academy; Affect; African; African American; Age; Algorithms; Alleles; American; Antinuclear Antibodies; Arthritis; Autoimmune Diseases; Autoimmune Process; Award; Blindness; Cataract; Cells; Characteristics; Child; Childhood; Chronic Childhood Arthritis; Chronic Disease; Clinical; Clinical Trials; Cohort Studies; Complement; Complication; Data; Databases; Detection; Development; Diagnosis; Disease; Early Diagnosis; Epidemiology; Etiology; European; Evaluation; Eye; Eye diseases; Flare; Genetic; Genetic Polymorphism; Genetic Risk; Glaucoma; Goals; Guidelines; HLA-B27 Antigen; HLA-DR1 Antigen; Health; Healthcare; Impact evaluation; Impairment; Inflammatory; Institution; Intervention; Lead; Master of Science; Measurement; Measures; Medical Records; Mentors; Monitor; Onset of illness; Ophthalmologist; Ophthalmology; Outcome; Outcome Measure; Outcomes Research; Pathogenesis; Patient-Focused Outcomes; Patients; Pediatrics; Play; Population; Predisposition; Prospective Studies; Proteins; Psyche structure; Quality of life; Questionnaires; Race; Research; Resources; Rheumatism; Rheumatology; Risk; Risk Factors; Risk Marker; Role; Scheme; Scientist; Screening procedure; Secondary to; Severities; Severity of illness; Social Functioning; Standardization; Stratification; Symptoms; Therapeutic; United States; Universities; Update; Uveitis; Variant; Vision; Vision Disorders; Visit; Visual; Visual Acuity; Visual impairment; aggressive therapy; base; career; clinical epidemiology; cohort; design; disability; disorder risk; experience; genetic risk factor; global health; illness length; improved; longitudinal course; prevent; professor; prospective; psychologic; psychosocial; rheumatologist; tertiary care; tool

DESCRIPTION (provided by applicant): 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. PUBLIC HEALTH RELEVANCE: Juvenile idiopathic arthritis-associated uveitis (JIA-U) is an inflammatory eye disease that affects 20% of children with JIA. This research will validate known risk factors and identify new risk markers of JIA-U, identifying populations of children at greatest risk for severe disability and blindness. It is anticipated that early detection of JIA-U, regular monitoring for eye disease and complications, and institution of appropriate therapy can significantly improve our children's quality of life and long-term outcomes.

描述（由申请人提供）：幼年特发性关节炎相关葡萄膜炎 (JIA-U) 是一种炎症性眼病，影响 20% 的 JIA 儿童，可导致白内障、青光眼、视力丧失和失明。目前的筛查指南旨在识别有幼年特发性眼病风险的儿童。然而，儿科风湿病学家和眼科医生提供葡萄膜炎全面筛查的能力有限，因为指南尚未更新以包括所有幼年关节炎亚型和其他已知的幼年特发性关节炎视力限制并发症的危险因素。幼年特发性关节炎和其他相关儿童自身免疫性疾病的筛查至关重要，因为早期葡萄膜炎检测可以预防视觉并发症并影响治疗管理。同样，在需要时进行早期积极治疗可能会避免严重的视力并发症。为了改善 JIA-U 儿童的健康结果和生活质量，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。通过识别葡萄膜炎易感性和严重程度的危险因素、标准化结果测量以及阐明 HLA 风险等位基因的重要性，早期准确地识别 JIA-U 将改善对 JIA-U 影响的评估和对 JIA-U 影响的理解。疾病的病因、发病机制和结果。同样，改进的风险分层方案可以减少不必要的眼科就诊，并帮助我们识别患有严重眼病和并发症的较高风险的儿童。本研究的目的是 1) 确定欧洲和非洲血统的 JIA 儿童的临床特征和 JIA-U 病程的差异，以确定葡萄膜炎的临床和遗传风险标记，2) 检查在对一组诊断为早期 JIA 的儿童进行为期 4 年的前瞻性研究，以确定哪些因素与葡萄膜炎的最终发展相关，并提供其纵向特征数据，3) 确定同时患有关节炎和葡萄膜炎对身体和视力的影响残疾，使用主观和客观测量的特定视觉生活质量和整体生活质量。我们希望利用综合的生物学、遗传学和心理社会方法，根据幼年特发性关节炎亚型制定更广泛适用的风险分层方案，并确定新的风险因素，以确定最有严重残疾和失明风险的儿童群体，以便采取干预措施来改善他们的整体成果。该候选人是埃默里大学的儿科风湿病学家和儿科助理教授。她获得了临床研究理学硕士学位。她致力于自身免疫性眼病儿童的临床结果研究。她组建了一支优秀的指导团队，在儿科风湿病学、儿科眼科、儿科流行病学、结果研究和测量方面拥有丰富的经验，这些都与她的目标相关。这个指导奖项将帮助她成为一名成功的独立临床科学家。 公共卫生相关性：幼年特发性关节炎相关葡萄膜炎 (JIA-U) 是一种炎症性眼病，影响 20% 的 JIA 儿童。这项研究将验证已知的风险因素并确定 JIA-U 的新风险标记，从而确定最有严重残疾和失明风险的儿童群体。预计早期发现 JIA-U、定期监测眼部疾病和并发症并采取适当的治疗可以显着改善儿童的生活质量和长期结果。