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• 批准号: 6913531
• 负责人: BARBARA Anne HABER
• 金额: $ 26.38万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-09-15 至 2009-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6913531
• 关键词: biliary atresia; clinical research; cooperative study; growth /development; hepatitis; human subject; human therapy evaluation; information systems; method development; newborn human (0-6 weeks); nutrition; patient oriented research

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.

描述（由申请人提供）： 该提案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，以进行胆道闭锁和新生儿肝炎的发病机制，自然史和病理生理学的调查。胆道闭锁和新生儿肝炎护理的进展受到许多因素的阻碍，包括难以在单一中心招募足够数量的患有这些诊断的儿童，所有标记为新生儿肝炎的条件的多样性以及缺乏国家数据库。在国家层面上，儿科肝脏研究的合作努力很少，这与加快儿科癌症或艾滋病进展的步伐不同。通过国家合作网络成功解决这些问题将取决于参与该联盟的各中心的科学领导和业务业绩。我们的建议由四个部分组成。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：综合数据库开发战略和说明。第三部分：一项为期三年的研究方案，以确定治疗，以改善儿童胆道闭锁的增长。第四部分：一项为期一年的研究方案，以检查锯齿状蛋白1作为儿童胆道闭锁的修饰基因。总之，CHOP团队拥有基础设施、设施、受试者可用性、临床研究者和科学研究者，可以成为胆道闭锁新生儿肝炎临床联盟的专职和富有成效的成员。