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.

项目摘要/摘要 克罗恩病（CD），一种严重的慢性炎症性肠病（IBD） 在小儿种群中被认为很少见，最近被认为是最重要的 影响儿童和青少年的慢性疾病，年轻人的发病率增加 不同的年龄。由于童年是一个身体，情感和社会成熟的时代，CD的发作 在童年时代可能会严重延迟成长，危害心理健康，负面影响可能 最后一生。在当前的小儿CD研究中，通常在疾病活动中评估疾病活性 治疗后的指定时间点，用于比较治疗和长期预测 结果。这些研究的发现基于患者反应的快照， 关于疾病进展的时间性质的有限信息。另外，离散分析 疾病活动无法提供有关治疗和治疗之间功能关系的见解 生长，这对于选择诱导和维持缓解的治疗至关重要 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 识别小儿CD疾病轨迹的模式（PCD），以及2）合并时间 疾病轨迹中包含的信息用于评估治疗和长期预测 术语成果。该项目的可交付成果将有助于全面理解 PCD病因，改善PCD患者的基于证据的治疗策略，可以 导致有关针对PCD优化护理标准的更好临床实践的变化 患者。我们将利用来自国家儿科PEDSNET的大儿科人群 学习卫生系统，以增强研究结果和影响。数据来自 该国八个最大的儿科卫生系统（包括波士顿儿童医院，包括 费城儿童医院和辛辛那提儿童医院医疗中心），覆盖 从2009年到2017年，总共有超过600万儿童和IBD诊断。 此外，我们的研究产生的新知识和开源软件将是 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。

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