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

儿童肝病研究和教育网络 (CHiLDREN) - UCSF (CC)

• 批准号: 8545792
• 负责人: PHILIP ROSENTHAL
• 金额: $ 20.93万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
• 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 2014-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8545792
• 关键词: Accounting; Advocacy; Alagille Syndrome; Ancillary Study; Biliary Atresia; California; Caring; Cause of Death; Centers for Disease Control and Prevention (U.S.); Child; Childhood; Cholestasis; Chronic; Clinic; Clinical; Clinical Trials; Collaborations; Communities; Cystic Fibrosis; Data; Databases; Development; Diagnosis; Disease; Education; Fibrosis; Genes; Image; Industry; Liver; Liver diseases; Longitudinal Studies; Medical; Methodology; Methods; New Agents; Outcome; Patient advocacy; Patients; Pediatric Hospitals; Pediatric Surgical Procedures; Phase; Play; Public Health; Publications; Research; Research Personnel; Resources; Role; Rotavirus; Safety; Site; Standardization; Steroids; Training; Transplantation; United States; Universities; Validation; children with cystic fibrosis; cohort; computerized data processing; cystic fibrosis patients; follow-up; group support; imaging modality; liver transplantation; novel diagnostics; programs; prospective; sample collection; screening; web site

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.

描述（由申请人提供）： UCSF目前是胆道闭锁研究联盟（BARC）和胆汁淤积性肝脏联盟（CLiC）的十个原始站点之一，该联盟正在继续，扩展和合并，以形成儿童肝病研究和教育网络（ChiLDREN）。新成立的ChiLDREN的具体目标可能包括但不限于：1）完成类固醇在胆道闭锁中的安全性和有效性临床试验。2)继续进行BARC和CLiC儿童前瞻性纵向研究，继续为辅助研究提供数据和生物标本，旨在为肝移植前后的儿童发现新的诊断、病因和治疗选择。3)继续进行囊性纤维化肝病（CFLD）的前瞻性纵向研究，旨在确定CF儿童肝脏疾病发展的预测因素以及CFLD儿童结局的预测因素。4)在CF患者中识别和验证肝脏疾病的非侵入性标志物，包括但不限于新的成像方式。5)治疗儿童胆汁淤积的新药的l/ll期研究。6)Alagille综合征和α-1-抗胰蛋白酶病修饰基因的研究7)在行业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者提供培训机会。9)继续为网络内的小型试点和示范项目提供支持。10）继续通过出版物和网站向科学界和非专业人士提供有关儿科肝病的教育。此外，在加州大学旧金山分校，我们特别计划继续研究HLA在胆道闭锁中的作用，与科拉拉多大学的Cara Mack博士合作。此外，为了确定轮状病毒是否在胆道闭锁中发挥病因学作用，我们计划将来自前BARC数据库和新ChiLDREN数据库的数据与美国CDC的轮状病毒活性数据进行比较。Bull博士计划开发快速可靠的PFIC诊断筛查方法。 相关性：BARC和CLiC的结合将增强临床信息不受阻碍流动的能力，提高效率，允许流程，数据和标本采集方法的标准化，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，并包括移植后儿童的随访。我们在加州大学旧金山分校的具体研究旨在帮助诊断和治疗胆道闭锁和PFIC。