METHODOLOGY CORE

方法论核心

• 批准号: 10466933
• 负责人: Maurizio Macaluso
• 金额: $ 30.68万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-16 至 2024-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10466933
• 关键词: Address; Adolescent; Affect; Age; American; Basic Science; Bayesian Analysis; Biological Availability; Biological databases; Biometry; Child; Childhood; Chronic; Chronic Childhood Arthritis; Clinical; Clinical Research; Clinical Trials; Clinical Trials Design; Collaborations; Communities; Complement; Complex; Data; Data Analyses; Data Science; Dermatomyositis; Development; Diagnosis; Diagnostic; Direct Costs; Dose; Ensure; Epidemiologist; Epidemiology; Epidermolysis Bullosa; Ethics; Family; Fever; Fibromyalgia; Funding; Future; Growth and Development function; Health; Health system; Healthcare; Human; Informatics; Infrastructure; Institution; Institutional Review Boards; Intervention; Knowledge; Learning; Link; Mediation; Medical center; Medicine; Methodology; Methods; Mission; Musculoskeletal; Musculoskeletal Diseases; National Institute of Arthritis and Musculoskeletal and Skin Diseases; Observational Study; Output; Patient Outcomes Assessments; Patient-Focused Outcomes; Patients; Pediatric Hospitals; Performance; Pharmaceutical Preparations; Phase; Phenotype; Prevention; Process; Production; Quality of life; Recurrence; Reproducibility of Results; Research; Research Design; Research Methodology; Research Personnel; Research Training; Resources; Rheumatism; Rheumatology; Safety; Sampling; Scheme; Science; Scleroderma; Statistical Data Interpretation; Syndrome; Systemic Lupus Erythematosus; Techniques; Technology; Testing; Therapeutic; Translating; Translational Research; Validation; base; clinical center; data acquisition; data integration; data management; data sharing; dissemination science; effective therapy; high standard; improved; innovation; mHealth; multidimensional data; novel strategies; precision medicine; programs; protocol development; response; sensor; skin disorder; tool

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.

抽象-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，维持一种 慢性病程，干扰生长、发育和生活质量。精准医疗 PMSKD的方法将有助于解决这些问题，并通过增加 生物数据库、患者表型鉴定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伦理和安全测试联系起来，并与 在医学实践中整合有效的治疗方法。CTR依赖于一组复杂的 方法，并需要方法论研究来支持其进展。针对RFA-AR-17- 002，由国家关节炎、肌肉骨骼和皮肤病研究所(NIAMS)发布 为了资助临床研究核心中心，辛辛那提儿童儿科肌肉骨骼和 风湿病创新核心中心(Portico)意在推动精准医学-- 面向PMKSD的CTR。该技术的性能、科学产出和患者结果 Portico研究基地将受益于协调努力和支持的基础设施 高标准的数据集成和共享、结果的再现性和高效使用 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 以及确保提供CTR所需证据的程序。我们将为PMSKD提供支持 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， 提供专家咨询和资源，以支持开展研究、统计分析和解释 研究结果；2)满足PMSKD的业务、信息学和数据处理需求 研究人员通过为数据采集提供最先进的资源和技术， 管理和利用；以及3)通过以下方式促进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：研究方法。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快 对PMSKD的科学发现和理解。它将带来新的治疗方法进行试验，翻译 以改善患者及其家人的健康和健康。