Mid-Atlantic BANH Consortium Site

描述(由申请人提供)： 这项建议的目的是证明费城儿童医院(CHOP)的医生和科学家小组有兴趣、基础设施和资源成为胆道闭锁新生儿肝炎临床联盟的杰出参与者，以调查胆道闭锁和新生儿肝炎的发病机制、自然病史和病理生理学。胆道闭锁和新生儿肝炎的护理进展受到许多因素的阻碍，包括难以在一个中心招募足够数量的确诊为此类疾病的儿童，各种疾病都被贴上新生儿肝炎的标签，以及缺乏国家数据库。与加快儿科癌症或艾滋病进展速度的努力不同，在国家一级的儿科肝脏研究方面几乎没有合作努力。能否通过国家协作网络解决这些问题，将取决于参与该联盟的各中心的科学领导能力和业务表现。我们的提案由四个部分组成。第一部分：描述费城儿童医院和宾夕法尼亚大学的医生和科学家团队的设施、资源和参与情况。第二节：开发综合数据库的战略和说明。第三部分：一项为期三年的研究方案，旨在确定改善胆道闭锁儿童生长发育的治疗方法。第四部分：一项为期一年的研究方案，检验锯齿状1作为胆道闭锁儿童的修饰基因。总而言之，CHOP团队拥有基础设施、设施、受试者可获得性、临床研究人员和科学研究人员，可以成为胆道闭锁新生儿肝炎临床联盟的敬业和富有成效的成员。

DESCRIPTION (provided by applicant): The aim of this proposal is to demonstrate that the group of physicians and scientists at Children's Hospital of Philadelphia (CHOP) have the interest, infrastructure and resources to be an outstanding participant in the Biliary Atresia Neonatal Hepatitis Clinical Consortium to conduct investigations of the pathogenesis, natural history and pathophysiology of Biliary Atresia and Neonatal Hepatitis. Progress in the care of Biliary Atresia and Neonatal Hepatitis is hampered by many factors including the difficulty in recruiting adequate numbers of children with these diagnoses at a single center, diversity of conditions that are all labeled neonatal hepatitis and the absence of a national database. There are few efforts at collaboration in pediatric liver research at the national level, unlike that instituted to accelerate the pace of progress in pediatric cancer or AIDS. Success in addressing these problems through a national collaborative network will depend on the scientific leadership and operational performance of the centers involved in the Consortium. Our proposal is composed of four sections. Section I: A description of the facilities, resources and participation of the team of physicians and scientists at Children's Hospital of Philadelphia and University of Pennsylvania. Section II: A strategy and description of the development of a comprehensive database. Section III: A three-year research protocol to identify therapy to improve growth in children with Biliary Atresia. Section IV: A one-year research protocol to examine Jagged 1 as a modifier gene in children with Biliary Atresia. In summary, the CHOP team has the infrastructure, facilities, subject availability, clinical investigators and scientific investigators to be a dedicated and productive member of the Biliary Atresia Neonatal Hepatitis Clinical Consortium.