A PROSPECTIVE DATABASE OF INFANTS WITH CHOLESTASIS

胆汁淤积婴儿的前瞻性数据库

• 批准号: 7374374
• 负责人: RONALD J. SOKOL
• 金额: $ 0.49万
• 依托单位: UNIVERSITY OF COLORADO DENVER
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-24 至 2007-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7374374
• 关键词: bile; blood; child rearing; children; cholestasis; diagnosis; hepatitis; information systems; liver; parents; tissues; urine

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The primary objectives of this research are to establish (1) a database containing clinical information and (2) a repository of blood and tissue samples from children with neonatal liver diseases such as biliary atresia and neonatal hepatitis to facilitate research in these important liver problems in children. This database and the specimens will be available for ancillary studies that will investigate the pathogenesis and natural history of biliary atresia and neonatal hepatitis or to evaluate patterns of cellular gene and protein expression in tissue specimens and plasma by viral, genomic and proteomic techniques. The study population will consist of infants, both male and female, with cholestasis who are less than or equal to 180 days old at the time of diagnosis at a Biliary Atresia Research Consortium (BARC) clinical site. In order to study the natural history, subjects will be followed until 10 years of age, liver transplantation or, for children without biliary atresia, until complete recovery off of all therapy. This study will: 1. collect detailed clinical and demographic information about each subject at enrollment and during follow up, 2. obtain and store blood and urine samples from the subject at diagnosis and during follow up, 3. obtain and store liver and biliary tissue and bile that are removed during diagnosis (i.e., biopsy) or at time of surgery or transplant and that are not needed for diagnostic purposes 4. collect demographic and medical history of parents at enrollment, and 5. obtain and store blood from the biological parents at enrollment. Samples of blood, urine, bile and tissue will be stored in repositories for future research. The data and biological specimens will be used for detailed study into the mechanisms and causes of liver problems in young children in order to try to better diagnose and manage these conditions. The subject will receive standard-of-care treatment and will not be restricted in type of treatment or from changes in treatment, such as newer treatments as they are developed. The subjects may not directly benefit from participation in this research, but in the future other children with similar problems may benefit from new information that may lead to better medical care.

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。本研究的主要目的是建立（1）包含临床信息的数据库和（2）来自患有新生儿肝脏疾病（如胆道闭锁和新生儿肝炎）的儿童的血液和组织样本的储存库，以促进对这些重要的儿童肝脏问题的研究。该数据库和标本将用于辅助研究，研究胆道闭锁和新生儿肝炎的发病机制和自然史，或通过病毒、基因组和蛋白质组学技术评价组织标本和血浆中细胞基因和蛋白质表达模式。研究人群将包括在胆道闭锁研究联盟（BARC）临床研究中心诊断时小于或等于180日龄的胆汁淤积婴儿（男性和女性）。为了研究自然史，将对受试者进行随访，直至10岁，接受肝移植，或者对于无胆道闭锁的儿童，直至所有治疗完全恢复。 本研究将：1.在招募时和随访期间收集关于每个受试者的详细临床和人口统计学信息，2.在诊断时和随访期间从受试者获得并储存血液和尿液样品，3.获得并储存在诊断期间取出的肝脏和胆管组织以及胆汁（即，活检）或在手术或移植时，并且不需要用于诊断目的4.收集入组时父母的人口统计学和病史，以及5.在入组时从亲生父母处获取并储存血液。 血液、尿液、胆汁和组织样本将储存在储存库中，以供未来研究。这些数据和生物标本将用于详细研究幼儿肝脏问题的机制和原因，以便更好地诊断和管理这些疾病。受试者将接受标准治疗，治疗类型或治疗变化（如开发的新治疗）不受限制。受试者可能不会直接受益于参与这项研究，但在未来，其他有类似问题的儿童可能会受益于新的信息，可能会导致更好的医疗保健。