Childhood Liver Disease Research and Education Network (CHiLDREN) - UCSF (CC)

描述(由申请人提供)： 加州大学旧金山分校目前是胆道闭锁研究联盟(BARC)和胆汁淤积性肝脏联盟(CLIC)的十个原始网站之一，该联盟正在继续、扩大和合并，以形成儿童肝病研究和教育网络(CHILDS)。新成立的儿童的具体目标可能包括但不限于：1)完成类固醇治疗胆道闭锁的安全性和有效性临床试验。2)继续对儿童进行BARC和CLIC前瞻性纵向研究，继续为旨在发现儿童肝移植前后新的诊断、病因和治疗选择的辅助研究提供数据和生物标本。3)囊性纤维性肝病(CFLD)前瞻性纵向研究的继续，目的是确定CFLD儿童肝病发展的预测因素以及CFLD儿童预后的预测因素。4)慢性肝病患者肝脏疾病非侵袭性标志物的识别和验证，包括但不限于新的成像方式。5)L期/Ⅱ期治疗儿童胆汁淤积症的新药研究。6)Alagille综合征和Alpha-1-Antrypsin病的修饰基因研究。7)在业界和患者倡导团体的支持下，扩大到包括儿童胆汁淤积症的其他疾病。8)继续为儿科肝病研究人员提供培训机会。9)继续支持该网络内的小型试点和示范项目。10)继续通过出版物和网站向科学界和非科学界提供有关儿科肝病的教育。此外，在加州大学旧金山分校，我们特别计划与科拉拉多大学的Cara Mack博士合作，继续研究人类白细胞抗原在胆道闭锁中的作用。此外，为了确定轮状病毒是否在胆道闭锁中起病因学作用，我们计划将来自以前的BARC数据库和新的儿童数据库的数据与美国疾病控制与预防中心的轮状病毒活动数据进行比较。布尔博士计划开发快速、可靠的诊断PFIC的筛查方法。 相关性：BARC和CLIC的结合将增强畅通无阻的临床信息流动的能力，允许更高的效率，允许过程、数据和样本收集方法的标准化，允许网络利用非专业组织提供的可用资源来扩大研究的疾病数量，并包括对儿童移植后的随访。我们在加州大学旧金山分校的专门研究旨在帮助诊断和治疗胆道闭锁和PFIC。

DESCRIPTION (provided by applicant): UCSF is currently one of the ten original sites in the Biliary Atresia Research Consortium (BARC) and the Cholestatic Liver Consortium (CLiC) which is being continued, expanded and merged to form the Childhood Liver Disease Research and Education Network (ChiLDREN). The specific objectives of the newly formed ChiLDREN could include but are not limited to: 1) Completion of the steroid safety and efficacy clinical trial in biliary atresia. 2) Continuation of the BARC and CLiC prospective longitudinal studies of children to continue to provide data and biospecimens for ancillary studies aimed at discovering new diagnostics, etiologic and treatment options for children both pre and post liver transplantation. 3) Continuation of the prospective longitudinal study of cystic fibrosis liver disease (CFLD) aimed at identifying predictors of development of liver disease in children with CF as well as predictors of outcome in children with CFLD. 4) Identification and validation of non-invasive markers of liver disease in CF patients including but not limited to new imaging modalities. 5) Phase l/ll study of new agents to treat cholestasis in children. 6) Study of modifier genes in Alagille Syndrome and Alpha-1-antrypsin disease. 7) Expansion to include other diseases of cholestasis in children with industry and patient advocacy group support. 8) Continue to provide training opportunities for investigators in pediatric liver disease. 9) Continue to provide support for small pilot and demonstration projects within the Network.10) Continue to provide education about pediatric liver diseases to the scientific and lay communities through publications and the website. In addition, at UCSF we specifically plan to continue to investigate the role of HLA in biliary atresia in collaboration with Dr. Cara Mack at the University of Colarado. Further, in order to determine if rotavirus plays an etiologic role in biliary atresia, we plan to compare data from the former BARC database and new ChiLDREN database with CDC data for rotavirus activity in the United States. Dr. Bull plans to develop fast and reliable screening methods for diagnosis of PFIC. Relevance: Combining BARC and CLiC will enhance the capacity for unhindered flow of clinical information, allow for greater efficiency, permit standardization of process, data, and specimen collection methodology, allow the network to take advantage of available resources offered by lay organizations to expand the number of diseases studied, and include follow-up of children after transplant. Our specific studies at UCSF aim to aid in diagnosis and treatment of biliary atresia and PFIC.