BILIARY ATRESIA RESEARCH CONSORTIUM (BARC): A PROSPECTIVE DATABASE OF INFANT

胆道闭锁研究联盟 (BARC)：婴儿前瞻性数据库

• 批准号: 8356666
• 负责人: SAUL J. KARPEN
• 金额: $ 3.26万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-12-01 至 2011-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8356666
• 关键词: 2 year old; Bile fluid; Biliary Atresia; Blood; Child; Clinical; Clinical Research; Databases; Development; Developmental Delay Disorders; Diagnosis; Disease; Epidemiology; Failure; Family; First Degree Relative; Funding; Genetic; Grant; Growth; Infant; Laboratories; Liver diseases; National Center for Research Resources; Operative Surgical Procedures; Outcome; Pathogenesis; Principal Investigator; Research; Research Infrastructure; Resources; Risk Factors; Source; Time; Tissue Sample; United States National Institutes of Health; Urine; abstracting; base; cost; disease natural history; genetic risk factor; prospective; repository; success; total measurement Bilirubin

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. ABSTRACT HYPOTHESIS 1. Environmental/epidemiological/genetic factors contribute to the pathogenesis of cholestatic liver diseases in children. 2. Ultrasonographic findings at diagnosis are specific for the diagnosis of biliary atresia. 3. Clinical parameters, such as laboratory values, (e.g. total bilirubin at 6 months post-surgery), the development of various clinical findings (e.g. ascities), are predictive of the evantual outcome of the Kasai Portoenterostomy by 2 years of age. Developmental delay can be predicted based upon serial clinical (e.g. growth failure) or laboratory values (e.g. total bilirubin). SPECIFIC AIMS Specific Aim 1: To establish a prospective database with demographic and clinical information about infants with cholestatic disease and their families. Specific Aim 2: To establish repositories for blood, urine, bile, and tissue samples from these children and their first-degree relatives. Specific Aim 3: To prospectively follow these children over time to characterize the natural history of the disease. Specicic Aim 4: To identify risk factors (such as, environmental, infectious and genetic risk factors) related to onset, to outcome and to the success of treatment(s) for the different cholestatic diseases, with special emphasis on biliary atresia.

这个子项目是利用资源的许多研究子项目之一。 由NIH/NCRR资助的中心拨款提供。对子项目的主要支持 子项目的首席调查员可能是由其他来源提供的， 包括美国国立卫生研究院的其他来源。为子项目列出的总成本可能 表示该子项目使用的中心基础设施的估计数量， 不是由NCRR赠款提供给次级项目或次级项目工作人员的直接资金。 摘要 假设 1.环境/流行病学/遗传因素导致儿童胆汁淤积性肝病。 2.诊断时超声表现对胆道闭锁的诊断具有特异性。 3.临床参数，如实验室数值(如术后6个月的总胆红素)、各种临床表现的发展(如腹水)，可预测Kasai门肠吻合术2岁前的预后。 发育迟缓可以根据一系列的临床数据(如生长障碍)或实验室数值(如总胆红素)来预测。 具体目标 具体目标1：建立一个关于胆汁淤积症婴儿及其家庭的人口学和临床信息的前瞻性数据库。 具体目标2：建立这些儿童及其一级亲属的血液、尿液、胆汁和组织样本的储存库。 具体目标3：前瞻性地跟踪这些儿童一段时间，以确定疾病的自然病史。 特殊目标4：确定与不同胆汁淤积性疾病的发病、转归和治疗成功有关的危险因素(如环境、感染和遗传危险因素)(S)，特别强调胆道闭锁。