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工作人员照顾约1000名患者，在过去的两年中，我们一直参与的BARC/CLiC/CFLD多中心研究小组研究了111名患有肝脏疾病的儿童。本ChiLDREN提案的目的是将约翰霍普金斯儿科肝脏中心的资源用于实现以下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BARC和CLiC肝病儿童发现新的诊断，病因和治疗方案。通过招募新的参与者，继续我们的研究中心参与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;这是一项研究，我们正在调查来自BA和遗传性胆汁淤积对照婴儿的血清（在BARC指导委员会批准的辅助研究中提供给我们），以证明对轮状病毒和轮状病毒感染的胆管细胞的体液免疫。3)继续参与囊性纤维化肝病（CFLD）的前瞻性纵向研究，旨在确定肝病发展和结局的预测因素。4)继续为儿科肝病研究者提供培训机会，例如我们建议在临床护理和CFLD相关研究中培训我们的一名儿科胃肠病学第一年研究员。她的项目是确定CFLD儿童肝纤维化的最佳非侵入性标志物。5)继续通过出版物和BARC和CLiC网站向科学界和非专业人士提供有关儿科肝病的教育，特别注重征求使用它的家庭对网站的反馈。6）继续我们已经建立的基础设施，以执行BARC，CLiC和CFLD。我们 致力于参与这个多中心研究小组，并相信通过这个小组， 有可能在改善患有这些严重儿科肝病的儿童的健康方面取得重大进展。 相关性：我们建议的相关性在于，我们相信实现改善我们正在研究的肝病儿童健康目标的最佳方法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的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脏疾病。

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.