Evaluation and treatment of enthesitis-related arthritis

应聘者描述(申请人提供)：应聘者是一名儿科风湿学初级研究员，接受过临床流行病学高级培训，重点研究慢性风湿病的药物流行病学和比较疗效，包括终末炎相关关节炎(ERA)，这是青少年特发性关节炎(JIA)的一种。候选人提出了一个全面的跨学科计划，将为她提供技能和经验，使她能够发展成为一名儿科风湿病的独立临床研究员。她的职业发展将由一个全面的合作调查小组指导，他们有着长期富有成效的合作历史。该项目将建立在她在MSCE培训和论文工作期间获得的药物流行病学和临床流行病学研究知识的基础上，并将使她为在患有各种形式的JIA的儿童中进行干预研究做好准备。贾樟柯是儿童时期最常见的风湿病，每1000名儿童中就有1到4名患病。Era是一个JIA类别，占JIA的10%-20%。医生治疗ERA是为了控制炎症和预防慢性葡萄膜炎、生长障碍、痉挛、关节破坏和功能受限等疾病。患有ERA的儿童的一部分将发展为强直性脊柱炎(AS)，这种情况的特征是脊柱和背部疼痛、僵硬，最终椎骨融合。观察性研究表明，只有不到20%的ERA儿童在确诊后五年内得到缓解。今天，提供和研究ERA治疗的重大挑战受到评估终端炎、疾病活动性和治疗反应的方法的限制。拟议的研究计划应对这些挑战，并将(1)评估体检发现鼻窦炎的准确性，(2)开发和验证综合ERA疾病活动指数，以及(3)比较ERA事件病例中替代治疗策略的有效性和安全性。这项研究将为未来ERA治疗算法的安全性、有效性和成本效益研究提供初步数据、假设和结果衡量标准。

DESCRIPTION (provided by applicant): The candidate is a junior investigator in pediatric rheumatology with advance training in clinical epidemiology and a research focus on pharmacoepidemiology and comparative effectiveness of chronic rheumatic disease, including enthesitis-related arthritis (ERA), which is a category of juvenile idiopathic arthritis (JIA). The candidate proposes a comprehensive interdisciplinary program that will provide her with the skills and experience for her development into an independent clinical investigator in pediatric rheumatology. Her career development will be guided by a comprehensive group of co-investigators with a long history of productive collaboration. The project will build upon the foundations of pharmacoepidemiology and clinical epidemiology research knowledge that she obtained during her MSCE training and thesis work and will prepare her to conduct intervention studies in children with all forms of JIA. JIA is the most common rheumatologic condition in childhood, affecting 1 to 4 per 1,000 children. ERA is a JIA category that accounts for 10-20% of JIA. Physicians treat ERA to control inflammation and to prevent morbidities such as chronic uveitis, growth disturbance, contracture, joint destruction, and functional limitation. A subset of children with ERA will progress to ankylosing spondylitis (AS), a condition characterized by spinal and back pain, stiffness, and eventual fusion of the vertebra. Observational studies suggest that less than 20% of children with ERA achieve remission within five years after diagnosis. Significant challenges to providing and studying ERA treatment today are limited by methods for assessing enthesitis, disease activity, and response to therapy. The proposed plan of research addresses these challenges and will (1) evaluate the accuracy of physical examination to detect enthesitis, (2) develop and validate a composite ERA disease activity index, and (3) compare the effectiveness and safety of alternative treatment strategies in incident cases of ERA. This research will provide the preliminary data, hypotheses, and outcome measures for future safety, efficacy, and cost- effectiveness studies of ERA treatment algorithms.