Comparative Effectiveness Research in Axial Spondyloarthritis

中轴型脊柱关节炎的比较疗效研究

基本信息

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
项目类别：
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

项目摘要

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中轴型脊柱关节炎比较有效性研究的独立临床研究者 (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。指导临床医生和患者制定个体化治疗计划的证据有限。治疗药物当前群体水平的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体治疗效果。 N-of-1 试验使用多重交叉来解决患者与治疗的相互作用并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得可以将日常临床护理转变为稳健、精心设计的 n-of-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试验期间。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物。在长期、务实、大规模的 N-of-1 试验中提供了研究不同药物在现实世界中的比较有效性的机会。用于队列识别和系统数据收集的创新试验设计和生物信息学工具将其中包括促进 axSpA 未来比较有效性研究的关键要素。该提案的总体目标是测试两种 NSAID 在 axSpA 中进行 N-of-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。拟议的系列 N-of-1 试验将为其他治疗药物的有效性比较提供框架 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。