METHODOLOGY CORE

方法论核心

• 批准号: 10680551
• 负责人: Maurizio Macaluso
• 金额: $ 30.24万
• 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/10680551
• 关键词: Acceleration; Address; Affect; Age; American; Basic Science; Bayesian Analysis; 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; Dedications; Development; Diagnosis; Diagnostic; Direct Costs; Dose; Ensure; Epidemiologist; Epidemiology; Epidermolysis Bullosa; Ethics; Family; Fever; Funding; Future; Growth; Health; Health system; Healthcare; Human; Informatics; Infrastructure; Institution; Institutional Review Boards; Intervention; Juvenile Dermatomyositis; Juvenile scleroderma; 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; Resources; Rheumatism; Rheumatology; Safety; Sampling; Scheme; Science; Statistical Data Interpretation; Syndrome; Systemic Lupus Erythematosus; Techniques; Technology; Testing; Therapeutic; Translating; Translational Research; Validation; base; clinical center; clinical training; data acquisition; data integration; data management; data sharing; disease phenotype; dissemination science; effective therapy; empowerment; high standard; improved; innovation; juvenile onset fibromyalgia; mHealth; multidimensional data; novel strategies; personalized intervention; 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.

摘要-方法论核心