The Johns Hopkins Pediatric Liver Center ChiLDREN Grant

描述(由申请人提供)： 约翰霍普金斯儿科肝脏中心的工作人员照顾了大约1000名患者，在过去两年中，我们一直参与的BARC/CLIC/CFLD多中心研究小组研究了111名患有肝病的儿童。这项儿童提案的目的是投入约翰霍普金斯儿科肝脏中心的资源，以实现以下6个具体目标：1)通过提供临床数据和生物标本，继续参与BARC和CLIC，为患有BARC和CLIC肝病的儿童发现新的诊断、病因和治疗方案。通过招募新参与者，继续我们的网站参与BARC START试验。2)扩大我们正在进行的轮状病毒在“人类胆道闭锁”中的作用的研究；在这项研究中，我们正在调查BA和遗传胆汁淤积控制婴儿的血清(在BARC指导委员会批准的辅助研究中向我们提供)，以寻找对轮状病毒和轮状病毒感染的胆管细胞的体液免疫的证据。3)继续参与囊性纤维性肝病(CFLD)的前瞻性纵向研究，旨在确定肝病发展和预后的预测因素。4)继续为儿科肝病研究人员提供培训机会，例如我们建议培训一名儿科胃肠病一年级研究员与CFLD相关的临床护理和研究。她的项目是确定CFLD儿童肝纤维化的最佳非侵入性标记物。5)继续通过出版物以及BARC和CLIC网站向科学界和非科学界提供有关儿科肝病的教育，特别注重征求使用该网站的家庭的反馈意见。6)继续我们为执行BARC、CLIC和CFLD而创建的现有基础设施。我们是 致力于参加这个多中心学习小组，并相信通过这个小组，它将是 有可能在改善患有这些严重儿科肝病的儿童的健康的目标方面取得重大进展。 相关性：我们建议的相关性在于，我们认为实现改善我们正在研究的肝病儿童健康目标的最佳方式是通过我们已经积极参与的这种多中心方法。我们对轮状病毒在人类胆道闭锁中作用的研究有可能提供更好、更快速的诊断，并最终导致更合理的免疫治疗和预防这种非常严重的婴儿肝病。

DESCRIPTION (provided by applicant): The Johns Hopkins Pediatric Liver Center Staff cares for ~1000 patients and, in the last two years for 111 children who have one of the liver diseases studied by the BARC/CLiC/CFLD multi-center study groups in which we have been participating. The purpose of this ChiLDREN proposal is to devote the resources of the Johns Hopkins Pediatric Liver Center to achieve the following 6 Specific Aims: 1) Continue our participation in BARC and CLiC by providing clinical data and biospecimens for discovery of new diagnostics, etiologic and treatment options for children with the BARC and CLiC liver diseases. Continue our site's involvement in the BARC START trial by enrolling new participants. 2) Expand our ongoing study of the Role of Rotavirus in "Human Biliary Atresia"; this is a study in which we are investigating sera from BA and genetic cholestatic control infants (provided to us in an Ancillary Study approved by the BARC Steering Committee) for evidence of humoral immunity to rotavirus and to rotavirus-infected cholangiocytes. 3) Continue our participation in the prospective longitudinal study of cystic fibrosis liver disease (CFLD) aimed at identifying predictors of development of liver disease and of outcome. 4) Continue to provide training opportunities for investigators in pediatric liver disease, as exemplified by our proposal to train one of our first year fellows in pediatric gastroenterology in clinical care and research related to CFLD. Her project is to identify the best noninvasive markers of liver fibrosis in children with CFLD. 5) Continue to provide education about pediatric liver diseases to the scientific and lay communities through publications and the BARC and CLiC websites, with a particular focus on soliciting feedback regarding the websites from the families who use it. 6) Continue the established infrastructure we have already created to perform BARC, CLiC, and CFLD. We are committed to participation in this multi-center study group and believe that through this group it will be possible to make major progress in the goal of improving the health of children with these serious pediatric liver diseases. Relevance: The relevance of our proposal is that we believe that the best way to accomplish the goal of improving the health of children with the liver diseases we are studying is via this multi-center approach in which we have already been actively participatin. Our studies of the Role of Rotavirus in Human Biliary Atresia have the potential of providing better, more rapid diagnostic and can ultimately lead to more rational immunotherapy and prevention of this very serious infantile liver disease.