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

抽象资源核 UNC CCCR的总体目标是加强临床研究的设计和实施 风湿性和肌肉骨骼疾病(RMD)，重点是骨关节炎(OA)，a 我们研究界的独特优势。表型鉴定与精密医学 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)预测表型所需的关键科学专门知识和分析资源 (按特定分组预测结果，例如“进步者”与“非进步者”)和 使用临床数据集的指令性表型(预测对特定干预的反应)， 2)就表型考虑向调查人员提供指导和建议(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。像许多慢性疾病一样，包括骨性关节炎在内的RMD并非如此 单一疾病，而不是由多种表型组成的异质性疾病 在潜在的病理生物学机制上有所不同。如果不考虑这些因素，这些 差异导致对旨在解决特定机制的干预措施的测试 在“错误的”病人身上采取行动。因此，RMD的成功治疗需要有针对性地进行， 并在特定亚群或亚型中进行测试，这些亚群或亚型具有不同的潜在病理生物学特征， 心理社会和疼痛机制，与精准医学的目标一致。这个 表型鉴定和精确医学资源核心将与 方法学核心提供的服务将极大地增强研究设计和添加 对正在进行的、已完成的和计划中的广泛临床研究的价值 以下目标：目标1.为预测提供咨询意见和分析服务 和指令性表型，供对定义新的或新的或 正在进行的骨性关节炎或其他RMD的临床研究，包括辅助研究和 规划阶段。目标2.通过以下方式应用表型和精确医学的创新策略 利用从我们的研究界精选的临床研究中收集的数据，增加价值 为这些研究提供便利，促进外部验证，并为今后的研究提供信息。目标3.鼓励 通过以下途径更广泛地实施RMD的表型和精准医学方法 教育和向地方、国家和国际研究传播方法论 社区。致力于表型鉴定和精确医学的资源核心将极大地 使CCCR支持的研究社区受益，产生重大影响 在临床RMD研究领域，并最终关于患有这些疾病的人的生活 慢性和致残性疾病。

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.