Characterizing Disease Trajectory for Improving Treatment in Pediatric Crohn's Disease

描述疾病轨迹以改善小儿克罗恩病的治疗

• 批准号: 10641954
• 负责人: Jing Huang
• 金额: $ 61.68万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-08-15 至 2025-05-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10641954
• 关键词: Adolescent; Adult; Affect; Aftercare; Age; Boston; Caring; Child; Child health care; Childhood; Chronic; Chronic Disease; Computer software; Crohn' s disease; Data; Development; Disease; Disease Outcome; Disease Progression; Disease remission; Emotional; Ensure; Etiology; Evaluation; Evidence based treatment; Face; Growth; Health; Health system; Healthcare; Healthcare Systems; Incidence; Inflammatory Bowel Diseases; Knowledge; Learning; Maintenance; Medical center; Mental Health; Methodology; Methods; Modeling; Monitor; Names; Nature; Outcome; Patients; Pattern; Pediatric Crohn' s disease; Pediatric Hospitals; Pediatric Research; Pediatrics; Personal Satisfaction; Phase; Philadelphia; Population; Principal Component Analysis; Remission Induction; Research; Risk Factors; Sample Size; Sampling; Scheme; Selection for Treatments; Series; Severities; Software Tools; Specific qualifier value; Subgroup; System; Time; Treatment outcome; Update; Visualization software; Youth; clinical decision-making; clinical practice; computerized tools; data quality; disease diagnosis; disease heterogeneity; disorder subtype; empowerment; health care delivery; improved; improved outcome; insight; novel; open source; optimal treatments; patient response; risk prediction; social; software development; tool; treatment comparison; treatment strategy

Project Summary/Abstract Crohn's disease (CD), a serious chronic inflammatory bowel disease (IBD) that was once considered rare in the pediatric population, is recently recognized as one of the most important chronic diseases that affect children and adolescents, with increasing incidence in youths of varying ages. As childhood is a time of physical, emotional, and social maturation, onset of CD in childhood can seriously delay growth, jeopardize mental health, and the negative impact may last a lifetime. In current pediatric CD research, disease activity is commonly evaluated at pre- specified time points post treatment for comparison of treatments and prediction of long-term outcomes. Findings from these studies are based on snapshots of patients' responses, with limited information on the temporal nature of disease progression. In addition, discrete analyses of disease activity fail to provide insights on functional relationship between treatment and growth, which is essential for selection of treatment to induce and maintain remission while optimizing the patient's growth and well-being. To fill this vital gap, we propose to 1) develop a series of statistical tools to characterize and identify patterns of disease trajectory of pediatric CD (PCD), and 2) incorporate the temporal information contained in disease trajectory for evaluation of treatments and prediction of long- term outcomes. The deliverables of this project will contribute to comprehensive understanding of PCD etiology, improvement of evidence-based treatment strategy for PCD patients, and may lead to changes toward better clinical practice regarding the criteria for optimized care for PCD patients. We will harness the large pediatric population from PEDSnet, a national pediatric learning health system, to empower the research findings and impacts. The data come from eight of the nation's largest pediatric health systems (including the Boston Children's Hospital, Children's hospital of Philadelphia and Cincinnati Children's Hospital Medical Center), covering more than 6 million children in total and 15 thousand with IBD diagnosis from 2009 to 2017. Furthermore, new knowledge and open-source software generated from our research will be integrated in real time with healthcare delivery systems to improve health care. Successful development of this proposed research has a promise of direct impacting the PCD patients in the PEDSnet and nationwide.

项目总结/文摘

项目成果

Learning Longitudinal Patterns and Subtypes of Pediatric Crohn Disease Treated With Infliximab via Trajectory Cluster Analysis.

• DOI: 10.1097/mpg.0000000000003370
• 发表时间: 2022-03-01
• 期刊: Journal of pediatric gastroenterology and nutrition
• 影响因子: 2.9
• 作者: Chen A;Stein R;Baldassano RN;Huang J
• 通讯作者: Huang J

Missing data matter: an empirical evaluation of the impacts of missing EHR data in comparative effectiveness research.

缺失数据很重要：对比较有效性研究中缺失 EHR 数据的影响进行实证评估。

• DOI: 10.1093/jamia/ocad066
• 发表时间: 2023
• 期刊: Journal of the American Medical Informatics Association : JAMIA
• 作者: Zhou,Yizhao;Shi,Jiasheng;Stein,Ronen;Liu,Xiaokang;Baldassano,RobertN;Forrest,ChristopherB;Chen,Yong;Huang,Jing
• 通讯作者: Huang,Jing