Clinical Center--Biliary Atresia Clinical Research Consortium

描述(由申请人提供)： 这项建议的长期目标是为胆道闭锁研究联盟做出贡献，以确保其成功地提高对胆道闭锁的病因、发病机制和疾病机制的了解，并改善受影响儿童的临床护理、诊断和管理。这是一个重要的项目，因为在美国，胆道闭锁每年影响大约600名儿童。其原因尚不清楚。目前还没有关于早期诊断或筛查策略的良好研究，这是改善患者护理所必需的。受影响儿童的医疗和外科护理并不理想。胆道闭锁是儿童肝移植的头号适应症，约占美国病例的50%。这种相对罕见的疾病的护理费用过高。更好地了解这种疾病应该会带来更好的护理、更好的结果和更低的护理成本。 这项提案的具体目标是#1充分参与胆道闭锁临床研究联盟的临床中心。这需要参与该联盟职能的发展，包括确定要收集的数据、要开展的项目以及如何传播信息；#2确定在赠款的五年期间中西部上部地区所有或几乎所有的胆道闭锁病例并将其输入该联盟的数据库，以确定疾病的发病率和重要的流行病学特征，如发病率的种族/民族、社会经济和性别差异、长期趋势以及与可识别的病毒流行的时间关系；#3检查选定的胆道闭锁患者队列中的预后指标；以及#4检验胆道闭锁是一种病毒诱导的免疫介导性疾病的假设。

DESCRIPTION (provided by applicant): The long-term objective of this proposal is to contribute to the Biliary Atresia Research Consortium to assure its success in improving the understanding of the etiology, pathogenesis, and disease mechanisms of biliary atresia and improving the clinical care, diagnosis and management of affected children. This is an important project because biliary atresia affects about 600 children each year in the US. Its cause is unknown. There are no good studies regarding early diagnosis or screening strategies, which are needed to improve patient care. The medical and surgical care of affected children is sub-optimal. Biliary atresia is the number one indication for pediatric liver transplantation, accounting for about 50% of cases in the US. The cost of care of this relatively rare disease is exorbitant. Better understanding of the disease should lead to improved care, better outcomes and reduced cost of care. The specific aims of this proposal are #1 To participate fully as a clinical center in the Biliary Atresia Clinical Research Consortium. This entails participation in the development of the functions of the consortium, including determining data to be collected, projects to be undertaken and how information is to be disseminated; #2 To identify and enter into the consortium database all or nearly all of the cases of biliary atresia in the upper mid-west region over the five year period of the grant to determine the incidence of the disease and important epidemiological characteristics, such as racial/ethnic, socioeconomic and gender differences in incidence, secular trends, and temporal relationship to identifiable viral epidemics; #3 To examine prognostic indicators in a select cohort of biliary atresia patients.; and #4 To examine the hypothesis that biliary atresia is a viral-induced, immune mediated disease.