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 这份提案将为王润生博士提供实现其长期目标所需的重要培训， 成为一名独立的临床研究者，进行中轴型脊柱关节炎的比较有效性研究 （axSpA），其总体研究计划旨在优化axSpA患者的临床管理 并改善axSpA的结果。 有限的证据可用于指导临床医生和患者制定个性化的治疗计划， 治疗药物的当前组水平有效性和安全性数据。传统的头对头 随机临床试验受到研究组数量少、成本高和无法测量的限制 个别治疗效果。N-of-1试验使用多交叉来解决患者-治疗相互作用， 测量个体对不同疗法的反应。生物医学信息学的最新进展使其 有可能将日常临床护理转变为稳健的精心设计的n-1试验。基于EHR的可计算 表型分析算法将改善队列识别并降低患者招募的复杂性 在这样的审判中。因此，我假设N-of-1试验将确定最有效的治疗方法， 用于个体症状控制的药剂。在长期、务实、大规模的N-of-1试验中， 提供了在真实的世界环境中研究不同药物的比较有效性的机会。 将采用创新的试验设计和生物信息学工具进行队列识别和系统性数据收集， 关键因素，其中包括促进未来在axSpA的比较有效性研究。 本提案的总体目标是测试两种NSAID在axSpA中进行1对1试验的可行性，并开发 信息学工具，以促进更大规模的此类试验的规划和实施。拟议 一系列N-of-1试验将为其他治疗药物在以下方面的比较有效性提供框架： axSpA患者结合基于EHR的队列识别工具，该试验设计将增加 个体患者的治疗精度，促进以患者为中心的研究和个性化医疗 在axSpA。