The UNC Core Center for Clinical Research: Phenotyping and Precision Medicine Resource Core

北卡罗来纳大学临床研究核心中心：表型分析和精准医学资源核心

• 批准号: 10017049
• 负责人: RICHARD F LOESER
• 金额: $ 26.53万
• 依托单位: UNIV OF NORTH CAROLINA CHAPEL HILL
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-11 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10017049
• 关键词: Academy; Active Learning; Address; African American; Ancillary Study; Area; Arthritis; Behavior Therapy; Biochemical; Biochemical Markers; Biomechanics; Chronic; Clinical; Clinical Markers; Clinical Research; Clinical Trials; Clinical and Translational Science Awards; Communication; Communities; Consult; Consultations; County; Coupled; Data; Data Set; Degenerative polyarthritis; Development; Disease; E-learning; Education; Educational Materials; Epidemiology; Evaluation; Exclusion Criteria; Future; Genetic Markers; Genomics; Goals; Health Services; Image; International; Intervention; Machine Learning; Measures; Methodology; Musculoskeletal Diseases; Observational Study; Outcome; Outcome Measure; Pain; Patients; Pharmacology; Phenotype; Proteomics; Publications; Research; Research Design; Research Personnel; Resources; Rheumatism; Services; Standardization; Subgroup; Supervision; Technology; Testing; Training; Validation; Vasculitis; Work; autoimmune vasculitis; clinical biomarkers; clinical center; clinical implementation; design; experience; imaging biomarker; improved; inclusion criteria; innovation; interest; medical schools; metabolomics; microbiome research; multidisciplinary; outcome prediction; precision medicine; predicting response; prognostic; psychosocial; socioeconomics; therapy design; unsupervised learning; webinar

ABSTRACT – RESOURCE CORE The overall goal of the UNC CCCR is to enhance the design and conduct of clinical studies in Rheumatic and Musculoskeletal Diseases (RMDs) with a focus on osteoarthritis (OA), a particular strength of our research community. The Phenotyping and Precision Medicine Resource Core will provide critical services to our research community that broadly include: 1) key scientific expertise and analytic resources needed for prognostic phenotyping (predicting outcomes by specific subgroups, e.g. “progressors” versus “non-progressors”) and prescriptive phenotyping (predicting response to specific interventions) using clinical datasets, and 2) guidance and advice to investigators on phenotypic considerations (such as choice of input and outcome variables) in order to enhance the design, implementation, and interpretation of new clinical studies and trials. Like many chronic conditions, RMDs, including OA, are not single diseases but rather heterogeneous conditions consisting of multiple phenotypes that differ in their underlying pathobiological mechanisms. If not taken into consideration, these differences result in the testing of interventions designed to address specific mechanisms of action in the “wrong” patients. Successful treatments for RMDs therefore need to be targeted to, and tested in, specific subgroups or subtypes that share distinct underlying pathobiological, psychosocial and pain mechanisms, consistent with the goal of precision medicine. The Phenotyping and Precision Medicine Resource Core will work in conjunction with the Methodology Core to provide services that will greatly enhance study design and add value to a wide spectrum of ongoing, completed, and planned clinical studies through the following aims: Aim 1. Provide consultative advice and analytical services for prognostic and prescriptive phenotyping for investigators interested in defining subgroups within new or ongoing clinical studies in OA or other RMDs, including ancillary studies and studies in the planning stages. Aim 2. Apply innovative strategies for phenotyping and precision medicine by leveraging data collected in selected clinical studies from our research community, adding value to those studies, facilitating external validation and informing future studies. Aim 3. Encourage broader implementation of phenotyping and precision medicine approaches for RMDs through education and dissemination of the methodologies to local, national, and international research communities. A resource core dedicated to phenotyping and precision medicine will greatly benefit the research community that will be supported by the CCCR, make a significant impact on the field of clinical RMD research, and ultimately on the lives of people suffering from these chronic and disabling conditions.

摘要 – 资源核心 UNC CCCR 的总体目标是加强临床研究的设计和实施 风湿病和肌肉骨骼疾病 (RMD)，重点关注骨关节炎 (OA)，这是一种 我们研究界的特殊实力。表型分析和精准医学 资源核心将为我们的研究社区提供广泛的关键服务 包括：1) 预后表型分析所需的关键科学专业知识和分析资源 （按特定亚组预测结果，例如“进步者”与“非进步者”）以及 使用临床数据集进行规定性表型分析（预测对特定干预措施的反应）， 2) 就表型考虑因素向研究者提供指导和建议（例如选择 输入和结果变量）以加强设计、实施和解释 新的临床研究和试验。与许多慢性病一样，RMD（包括 OA）并不 单一疾病，而是由多种表型组成的异质病症 其潜在的病理生物学机制有所不同。如果不考虑这些 差异导致对旨在解决具体机制的干预措施进行测试 对“错误”的患者采取行动。因此，成功治疗 RMD 需要针对： 并在具有不同潜在病理生物学特征的特定亚组或亚型中进行测试， 心理社会和疼痛机制，与精准医学的目标一致。这 表型和精准医学资源核心将与 方法论核心提供的服务将极大地增强研究设计并增加 通过以下方式对正在进行的、已完成的和计划的临床研究产生广泛的价值 目标如下： 目标 1. 提供预后咨询建议和分析服务 以及为有兴趣定义新的或新的亚组的研究人员提供规范的表型分析 正在进行的 OA 或其他 RMD 临床研究，包括辅助研究和 规划阶段。目标 2. 应用创新策略进行表型分析和精准医学 利用我们研究界选定的临床研究中收集的数据，增加价值 这些研究，促进外部验证并为未来的研究提供信息。目标 3. 鼓励 通过更广泛地实施 RMD 的表型分析和精准医学方法 对地方、国家和国际研究方法进行教育和传播 社区。致力于表型分析和精准医学的资源核心将极大地促进 使 CCCR 支持的研究界受益，产生重大影响 关注 RMD 临床研究领域，并最终关注患有这些疾病的人们的生活 慢性和致残疾病。