The UNC Core Center for Clinical Research: Methodologic Core

北卡罗来纳大学临床研究核心中心：方法学核心

• 批准号: 10017045
• 负责人: TODD A SCHWARTZ
• 金额: $ 24.61万
• 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/10017045
• 关键词: Advocate; Affect; American; Area; Arthritis; Big Data Methods; Bioinformatics; Biometry; Caring; Clinical; Clinical Research; Clinical Trials; Collaborations; Communities; Complex; Consultations; Degenerative polyarthritis; Development; Disease; Disease Progression; Education; Educational Materials; Epidemiologic Methods; Fostering; Foundations; Goals; Heterogeneity; Image; Individual; Infrastructure; International; Lead; Learning; Logistics; Machine Learning; Mentors; Methodology; Methods; Modeling; Musculoskeletal Diseases; North Carolina; Pain; Patient Selection; Patients; Phase; Phenotype; Positioning Attribute; Prevalence; Process; Public Policy; Research; Research Methodology; Research Personnel; Resources; Rheumatism; Sample Size; Schools; Selection Criteria; Seminal; Services; Source; Statistical Methods; Supervision; Training; Universities; base; care outcomes; clinical center; data harmonization; design; disability; epidemiology study; health disparity; high dimensionality; imaging biomarker; improved; innovation; learning outcome; medical schools; multidisciplinary; optimal treatments; precision medicine; recruit; research study; statistical service; therapy development; training opportunity; treatment effect; treatment guidelines; treatment response; treatment strategy; trial design; unsupervised learning; working group

ABSTRACT – METHODOLOGY CORE Rheumatic and musculoskeletal diseases (RMDs), including osteoarthritis (OA), are major sources of pain and disability worldwide, yet significant gaps persist in our understanding of disease mechanisms and optimal treatment strategies for many RMDs. There is a critical need for high quality, advanced studies that will meaningfully improve our understanding of RMDs, particularly phenotypes that predict disease progression and treatment response. The Methodology Core of our University of North Carolina (UNC) Core Center for Clinical Research (CCCR) is uniquely equipped to provide a comprehensive suite of resources that will optimize clinical RMD studies locally, nationally and internationally. Our Methodology Core includes researchers with expertise in biostatistics and bioinformatics, epidemiological studies, clinical trials, training and mentoring, health disparities, intervention development, dissemination and implementation, along with RMD-specific expertise in areas including imaging, biomarkers, and treatment guidelines. We have a long, successful track record of designing and conducting seminal clinical RMD studies (particularly in OA), developing junior investigators into independent researchers, fostering national/international collaborations, delivering methodological training to the RMD research community, leading national and international RMD- focused work groups and communicating, advocating, and influencing public policy. Building and expanding on our proven processes and established infrastructure, the Methodology Core will continue to serve the RMD research community by achieving three specific aims. First, we will support the development and application of advanced and innovative statistical methodologies (e.g., supervised and unsupervised machine learning, outcome-weighted learning, Markov state modeling, high dimensional low sample size methods, heterogeneity of treatment effects) to clinical RMD studies; this will include support for our Phenotyping and Precision Medicine Core. Second, we will provide comprehensive, rigorous multidisciplinary methodological resources that support and advance clinical RMD research within the CCCR base, as well as through collaborative efforts locally, nationally and internationally. Third, we will deliver state-of-the-art educational materials and training that prepare researchers, both locally and nationally/internationally, to conduct high quality, innovative clinical studies on RMDs. Collectively, these aims will provide services to a large, international clinical RMD research community, resulting in studies that lead to significant improvements in care and outcomes for the many patients living with RMDs.

摘要-方法论核心 风湿性和肌肉骨骼疾病（RMD），包括骨关节炎（OA），是疼痛和关节炎的主要来源。 残疾，但我们对疾病机制和最佳治疗方法的理解仍然存在重大差距。 针对多种RMD的治疗策略。迫切需要高质量的高级研究， 有意义地提高我们对RMD的理解，特别是预测疾病进展的表型 和治疗反应。我们的北卡罗来纳州大学（北卡罗来纳州）核心中心的方法论核心， 临床研究（CCCR）是唯一配备提供一套全面的资源， 优化本地、国内和国际的临床RMD研究。我们的核心方法包括 具有生物统计学和生物信息学、流行病学研究、临床试验、培训等专业知识的研究人员 和指导，健康差距，干预措施的制定，传播和实施，沿着 RMD在成像、生物标志物和治疗指南等领域的专业知识。我们有一个漫长的， 设计和开展开创性临床RMD研究（特别是OA）的成功记录， 将初级调查员培养成独立的研究人员，促进国家/国际合作， 为RMD研究界提供方法论培训，领导国家和国际RMD- 重点工作组和沟通，倡导和影响公共政策。建立和扩大 我们已经证明的过程和已经建立的基础设施，方法论核心将继续服务于RMD 研究团队通过实现三个具体目标。一是支持开发和应用 先进和创新的统计方法（例如，监督和无监督机器学习， 结果加权学习，马尔可夫状态建模，高维低样本方法，异质性 的治疗效果），以临床RMD研究;这将包括支持我们的表型和精度 药核。第二，我们将提供全面、严谨的多学科方法资源 支持和推进CCCR基地内的临床RMD研究，以及通过合作努力 在当地、国内和国际上。第三，我们将提供最先进的教育材料和培训 为本地和国家/国际研究人员做好准备，以进行高质量的创新临床研究， RMD的研究。总的来说，这些目标将为大型国际临床RMD研究提供服务 社区，导致研究，导致显着改善护理和成果，为许多 RMD患者。