METHODOLOGY CORE

摘要-方法论核心 儿童风湿性和非炎症性肌肉骨骼疾病（PMSKD）影响830万人 18岁以下的美国人（11%）。PMSKD很难诊断和治疗， 慢性过程和干扰生长，发育和生活质量。一种精确的医学 PMSKD的方法将有助于解决这些问题，并通过增加 生物数据库、患者表型分析技术和统计工具的可用性， 分析高维数据。临床和转化研究（CTR）至关重要 将基础科学的进步与人类的道德和安全测试联系起来， 在医学实践中整合有效的治疗。CTR依赖于一系列复杂的 方法，并需要方法学研究，以支持其进展。根据RFA-AR-17- 002，由国家关节炎、肌肉骨骼和皮肤疾病研究所（NIAMS）发布 资助辛辛那提儿童儿科肌肉骨骼和 RheumaTology创新核心中心（PORTICO）旨在促进精准医疗- 面向PMKSD的CTR。的性能，科学产出和病人的结果， PORTICO研究基地将受益于协调努力和维护 数据集成和共享的高标准、结果的可重复性以及 患者数据和样本。PORTICO方法核心（MC）将提供先进的信息学， 临床试验设计和数据管理技术方面的统计学和流行病学专业知识 和流程，以确保制作CTR所需的证据。我们将1）赋予PMSKD权力 研究人员拥有最先进的研究设计和方案开发，高效的研究启动， 专家建议和资源，以支持研究开展、统计分析和解释 研究结果; 2）满足PMSKD的操作，信息学和数据操作需求 研究人员通过提供最先进的数据采集资源和技术， 管理和利用;和3）促进PMSKD的临床研究和培训， 发展、应用和传播先进的流行病学、生物统计学和信息学 方法.我们的专业知识、基础设施、技术、机构支持以及我们的方法， 促进方法学家和临床研究人员之间的合作将加速 对PMSKD的科学发现和理解。它将带来新的治疗试验，翻译 改善患者及其家人的健康状况。

ABSTRACT – METHODOLOGY CORE Pediatric rheumatic and non-inflammatory musculoskeletal diseases (PMSKD) affect 8.3 million Americans under the age of 18 (11%). PMSKD are difficult to diagnose and treat, maintain a chronic course and interfere with growth, development and quality of life. A precision medicine approach to PMSKD will help address these problems and is made feasible by increasing availability of biologic databases, patient phenotyping techniques and statistical tools for analyzing high-dimensional data. Clinical and Translational Research (CTR) is critically important for linking advances in basic science with ethical and safe testing in humans, and with the integration of effective treatments in the practice of medicine. CTR relies on a complex array of methods, and needs methodologic research to support its progress. In response to RFA-AR-17- 002, issued by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) to fund Core Centers for Clinical Research, Cincinnati Children's Pediatric musculOskeletal & RheumaTology Innovation COre center (PORTICO) intends to promote precision medicine- oriented CTR for PMKSD. The performance, scientific output and patient outcomes of the PORTICO research base will benefit from an infrastructure that coordinates efforts and upholds high standards for data integration and sharing, reproducibility of results, and efficient use of patient data and samples. The PORTICO Methods Core (MC) will provide advanced informatics, statistical and epidemiological expertise in clinical trial design, and data management technology and processes to ensure the production of evidence needed by CTR. We will 1) Empower PMSKD researchers with state-of-the art study design and protocol development, efficient study start-up, expert advice and resources to support study conduct, statistical analysis and interpretation of study results; 2) Meet the operational, informatics, and data manipulation needs of PMSKD researchers by providing state-of-the art resources and technology for data acquisition, management and utilization; and 3) Promote clinical research and training in PMSKD by developing, applying and disseminating advanced epidemiologic, biostatistical and informatics methods. Our expertise, infrastructure, technology, institutional support, and our approach to promoting the collaboration between methodologists and clinical researchers will accelerate scientific discovery and understanding of PMSKD. It will bring new treatments to trial, translating into improved health and wellness for patients and their families.