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: 10414884
• 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 2024-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10414884
• 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 Progression; Disease remission; Emotional; Ensure; Etiology; Evaluation; Evidence based treatment; Face; Growth; Health; Health system; Healthcare; Healthcare Systems; Incidence; Inflammatory Bowel Diseases; Knowledge; Lead; 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; Prediction of Response to Therapy; Principal Component Analysis; Remission Induction; Research; Risk; 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; base; clinical decision-making; clinical practice; computerized tools; data quality; disease diagnosis; disease heterogeneity; health care delivery; improved; improved outcome; insight; novel; open source; optimal treatments; patient response; 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.

项目概要/摘要 克罗恩病 (CD) 是一种严重的慢性炎症性肠病 (IBD)，曾经 被认为在儿科人群中罕见，最近被认为是最重要的疾病之一 影响儿童和青少年的慢性疾病，青少年发病率不断上升 不同的年龄。由于童年是身体、情感和社会性成熟的时期，CD 的发病 儿童时期可能会严重延迟生长，危害心理健康，其负面影响可能 持续一生。在当前的儿科 CD 研究中，疾病活动度通常在治疗前进行评估。 治疗后的指定时间点，用于治疗比较和长期预测 结果。这些研究的结果基于患者反应的快照， 关于疾病进展的时间性质的信息有限。此外，离散分析 疾病活动的分析未能提供有关治疗和治疗之间功能关系的见解 生长，这对于选择治疗方法以诱导和维持缓解至关重要 优化患者的成长和福祉。 为了填补这一重要空白，我们建议 1）开发一系列统计工具来表征和 确定儿科 CD (PCD) 的疾病轨迹模式，2) 将时间纳入 疾病轨迹中包含的信息，用于评估治疗和预测长期疾病 期限结果。该项目的可交付成果将有助于全面理解 PCD 病因学的研究，改进 PCD 患者的循证治疗策略，并可能 导致关于 PCD 优化护理标准的更好临床实践的变化 患者。我们将利用来自 PEDSnet 的大量儿科人口，PEDSnet 是一个国家儿科网络 学习健康系统，赋予研究成果和影响力。数据来自 全国最大的八个儿科医疗系统（包括波士顿儿童医院、 费城儿童医院和辛辛那提儿童医院医疗中心），涵盖 2009年至2017年，总共有超过600万儿童，其中有1.5万名儿童被诊断为IBD。 此外，我们的研究产生的新知识和开源软件将被 与医疗保健提供系统实时集成，以改善医疗保健。成功的 这项拟议研究的发展有望直接影响 PCD 患者 PEDSnet 和全国范围内。