Evaluation and treatment of enthesitis-related arthritis

附着点炎相关性关节炎的评估和治疗

基本信息

批准号：
8655793
负责人：
Pamela Fitch Weiss
金额：
$ 11.65万
依托单位：
CHILDREN'S HOSP OF PHILADELPHIA
依托单位国家：
美国
项目类别：
财政年份：
2011
资助国家：
美国
起止时间：
2011-05-01 至 2016-04-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8655793
关键词：
6 year old Accounting Address Adult Affect Algorithms Ankylosing spondylitis Antirheumatic Agents Arthritis Back Pain Categories Child Childhood Chronic Chronic Childhood Arthritis Clinical Clinical Investigator Collaborations Combined Modality Therapy Consensus Contracture Data Detection Development Diagnosis Disease Disease remission Effectiveness Epidemiologic Studies Etanercept Evaluation Expert Opinion Family history of Foundations Future Gold Growth HLA-B27 Antigen Inflammation Inflammatory Intervention Studies Joints Knowledge Leflunomide Licensing Ligaments Measures Methods Methotrexate Morbidity - disease rate Neoadjuvant Therapy Non-Steroidal Anti-Inflammatory Agents Observational Study Osteopenia Outcome Outcome Measure Pain Pharmaceutical Preparations Pharmacoepidemiology Physical Examination Physicians Predictive Value Psoriatic Arthritis Recording of previous events Research Research Personnel Rheumatism Rheumatoid Arthritis Rheumatology Safety Sensitivity and Specificity Spinal Sulfasalazine Swelling Symptoms Techniques Tendon structure Time Training Treatment Protocols Ultrasonography Uveitis Work adalimumab alternative treatment base bone calcification career development clinical Diagnosis clinical epidemiology clinical practice clinically significant cohort comparative effectiveness compare effectiveness cost effectiveness expectation experience improved indexing infliximab joint destruction male pressure prevent primary outcome programs response skills soft tissue spine bone structure tool treatment strategy

项目摘要

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.

描述（由申请人提供）：候选人是儿科风湿病学的初级研究员，接受过临床流行病学的高级培训，研究重点是药物流行病学和慢性风湿病的比较有效性，包括附着点炎相关关节炎（ERA），这是一种幼年特发性关节炎（JIA）。候选人提出了一个全面的跨学科计划，该计划将为她提供发展成为儿科风湿病学独立临床研究者的技能和经验。她的职业发展将由具有悠久富有成效合作历史的综合性联合研究人员团队指导。该项目将建立在她在 MSCE 培训和论文工作中获得的药物流行病学和临床流行病学研究知识的基础上，并使她为对患有各种形式的幼年特发性关节炎的儿童进行干预研究做好准备。 JIA 是儿童时期最常见的风湿病，每 1,000 名儿童中就有 1 至 4 名患有 JIA。 ERA是JIA的一个类别，占JIA的10-20%。医生治疗 ERA 是为了控制炎症并预防慢性葡萄膜炎、生长障碍、挛缩、关节破坏和功能限制等疾病。一部分患有 ERA 的儿童会发展为强直性脊柱炎 (AS)，这种疾病的特征是脊柱和背部疼痛、僵硬，并最终导致椎骨融合。观察性研究表明，只有不到 20% 的 ERA 儿童在诊断后五年内获得缓解。如今提供和研究 ERA 治疗面临的重大挑战受到评估附着点炎、疾病活动性和治疗反应的方法的限制。拟议的研究计划解决了这些挑战，并将（1）评估体格检查检测附着点炎的准确性，（2）开发和验证综合 ERA 疾病活动指数，以及（3）比较替代治疗策略的有效性和安全性ERA 事件案例。这项研究将为 ERA 治疗算法的未来安全性、有效性和成本效益研究提供初步数据、假设和结果测量。