The Johns Hopkins Pediatric Liver Center ChiLDREN Grant

约翰·霍普金斯小儿肝脏中心 ChiLDREN 资助

• 批准号: 8012547
• 负责人: KATHLEEN SCHWARZ
• 金额: $ 15万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-02-20 至 2012-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8012547
• 关键词: Acute; Ancillary Study; Antibodies; Audiology; Biliary Atresia; Binding; Caring; Cell Line; Child; Child health care; Childhood; Clinical Data; Commit; Communities; Cystic Fibrosis; DEXA; Development; Diagnosis; Diagnostic; Disease; Disease Outcome; Education; Enrollment; Epitopes; Exhibits; Family; Feedback; Frequencies; Gastroenterology; Genetic; Goals; Grant; Human; Humoral Immunities; Immunotherapy; Infant; Investigational Drugs; Lead; Liver; Liver Fibrosis; Liver diseases; Longitudinal Studies; Measures; Molecular Mimicry; Mothers; Nutritionist; Outcome; Outpatients; Participant; Patients; Peptides; Phage Display; Pharmacy facility; Prevention; Publications; Research; Research Infrastructure; Research Personnel; Resources; Role; Rotavirus; Rotavirus Infections; Scanning; Sensitivity and Specificity; Serum; Site; Study of serum; Testing; Training; Transplant Surgeon; Validation; Visit; cDNA Library; children with cystic fibrosis; cholangiocyte; clinical care; improved; infancy; liver transplantation; neurobehavioral; novel diagnostics; prospective; radiologist

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.

约翰·霍普金斯（Johns Hopkins）小儿肝脏中心工作人员关心约1000名患者，在过去的两年中，有111名患有BARC/CLIC/CLIC/CFLD多中心研究小组研究的肝病之一的儿童。这个孩子的建议的目的是投入约翰·霍普金斯小儿肝脏中心的资源，以实现以下6个具体目标：1）继续我们参与BARC和CLIC，通过提供临床数据和生物测量来发现新的诊断，病因学和治疗方法，为BARC和CLIC LIVER疾病提供新的诊断和治疗方案。通过注册新参与者，继续我们网站参与BARC开始审判。 2）扩大我们对轮状病毒在“人类胆道闭锁”中的作用的持续研究；这是一项研究，我们正在研究BA和遗传胆固醇对照婴儿的血清（在由BARC指导委员会批准的辅助研究中提供给我们），以证明对轮状病毒的体液免疫和转身病毒感染的胆管造细胞。 3）继续我们参与囊性纤维化疾病（CFLD）的前瞻性纵向研究，旨在识别肝病发展和预后的预测指标。 4）继续为小儿肝病的研究人员提供培训机会，这是我们的提议训练与CFLD有关的临床护理和研究的小儿胃肠病学研究者之一的提议。她的项目是确定CFLD儿童中肝纤维化的最佳无创标志物。 5）继续通过出版物以及BARC和CLIC网站向科学和外行社区提供有关儿科肝病的教育，特别着眼于征求有关使用该网站的网站的反馈。 6）继续我们已经创建的已建立的基础架构来执行BARC，CLIC和CFLD。我们是 致力于参加这个多中心研究小组，并相信通过该小组 在改善这些严重的小儿肝病的儿童健康的目标方面取得了重大进展。