Comparative Effectiveness Research in Axial Spondyloarthritis

中轴型脊柱关节炎的比较疗效研究

• 批准号: 9892603
• 负责人: Runsheng Wang
• 金额: $ 17.05万
• 依托单位: COLUMBIA UNIVERSITY HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-08-15 至 2021-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9892603
• 关键词: Address; Adoption; Adult; Affect; Algorithms; Ankylosing spondylitis; Biological; Chronic; Clinical Investigator; Clinical Management; Code; Combined Modality Therapy; Comparative Effectiveness Research; Coupled; Crossover Design; Data; Data Collection; Development; Diagnostic; Disease; Disease remission; Effectiveness; Electronic Health Record; Elements; Eligibility Determination; Enrollment; Ensure; Family; Future; Goals; Head; Individual; Indomethacin; Inflammatory; Interleukin-17; Janus kinase; Life; Measures; Methods; Non-Steroidal Anti-Inflammatory Agents; Observational Study; Outcome; Participant; Patient Outcomes Assessments; Patient Recruitments; Patients; Pharmaceutical Preparations; Phenotype; Population; Precision therapeutics; Prediction of Response to Therapy; Predictive Value; Process; Public Health; Randomized Clinical Trials; Randomized Controlled Trials; Reporting; Research; Safety; Sensitivity and Specificity; Series; Spondylarthritis; Symptoms; TNF gene; Testing; Therapeutic Agents; Training; Treatment Efficacy; United States; Validation; Vertebral column; arm; base; bioinformatics tool; biomedical informatics; celecoxib; clinical care; clinical practice; cohort; comorbidity; comparative effectiveness; comparative effectiveness study; comparative efficacy; computable phenotypes; cost; data tools; design; evidence base; improved; individual patient; individual response; individualized medicine; informatics tool; inhibitor/antagonist; innovation; kinase inhibitor; novel; patient oriented; personalized medicine; phenotyping algorithm; programs; reduce symptoms; response; tool; treatment effect; treatment planning; treatment strategy; trial design

PROJECT ABSTRACT This proposal will provide Dr. Runsheng Wang with the vital training needed to achieve her long-term goal of becoming an independent clinical investigator in comparative effectiveness research in axial spondyloarthritis (axSpA), with an overall research program aimed at optimizing clinical management of patients with axSpA and improving axSpA outcomes. Limited evidence is available to guide clinicians and patients to make an individualized treatment plan based on current group level effectiveness and safety data of therapeutic agents. Traditional head-to-head randomized clinical trials are limited by small number of study arms, high cost, and inability to measure individual treatment effects. N-of-1 trials use multi-crossover to address patient-treatment interaction and to measure individual responses to different therapies. Recent advances in biomedical informatics make it possible to transform everyday clinical care to robust well designed n-of-1 trials. An EHR-based computable phenotyping algorithm would improve cohort identification and reduce the complexity of patient recruitment during such trials. Therefore, I hypothesize that N-of-1 trials would identify the most effective therapeutic agents for symptom control in individuals. In the long-term, pragmatic, large scale N-of-1 trials would provide the opportunity to study comparative effectiveness of different drugs in the real world setting. Innovative trial design and bioinformatics tools for cohort identification and systemic data collection will be key elements, among others, in facilitating future comparative effectiveness research in axSpA. The overall goal of this proposal is to test the feasibility of N-of-1 trials of two NSAIDs in axSpA and to develop informatics tools to facilitate the planning and implementation of such trials in a larger scale. The proposed series of N-of-1 trials will provide a framework for comparative effectiveness of other therapeutic agents in patients with axSpA. Coupled with an EHR-based cohort identification tool, this trial design will increase therapeutic precision in individual patients and promote patient-centered research and personalized medicine in axSpA.

项目摘要 该建议将为Runsheng Wang博士提供实现她长期目标所需的重要培训 成为轴向脊柱炎的比较有效性研究中的独立临床研究者 （AXSPA），旨在优化AXSPA患者的临床管理的整体研究计划 并改善AXSPA结果。 有限的证据可指导临床医生和患者制定基于个性化治疗计划 在当前的小组水平效力和治疗剂的安全数据上。传统的头对头 随机临床试验受到少量研究臂，高成本和无法测量的限制 个体治疗效果。 N-1-1试验使用多切换来解决患者治疗的互动和 测量个人对不同疗法的反应。生物医学信息学的最新进展使得 可能会将日常临床护理转变为健壮的精心设计的N-OF-1试验。基于EHR的可计算 表型算法将改善队列鉴定并降低患者招募的复杂性 在此类试验中。因此，我假设N-1-1试验将确定最有效的治疗方法 个体症状控制的代理。从长期的，务实的大规模N-1-1-1试验中 提供了研究在现实世界环境中不同药物的比较有效性的机会。 用于队列识别和全身数据收集的创新试验设计和生物信息学工具将是 关键要素，以及促进AXSPA的未来比较有效性研究。 该提案的总体目标是测试AXSPA中两个NSAID的N-1-1试验的可行性，并开发 信息学工具，以促进大规模计划和实施此类试验。提议 一系列N-1-1试验将为其他治疗剂在相对有效性中提供一个框架 AXSPA患者。加上基于EHR的同类识别工具，该试验设计将增加 个别患者的治疗精度，并促进以患者为中心的研究和个性化医学 在Axspa中。