The Johns Hopkins Pediatric Liver Center ChiLDREN Grant

约翰·霍普金斯小儿肝脏中心 ChiLDREN 资助

基本信息

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
项目类别：
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

项目摘要

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·霍普金斯大学儿科肝脏中心的工作人员护理了大约 1000 名患者，在过去两年中，还护理了 111 名患有我们参与的 BARC/CLiC/CFLD 多中心研究小组所研究的肝脏疾病之一的儿童。 ChiLDREN 提案的目的是投入约翰霍普金斯小儿肝脏中心的资源来实现以下 6 个具体目标： 1) 继续参与 BARC 和 CLiC，提供临床数据和生物样本以发现新的诊断、病因和治疗方法患有 BARC 和 CLiC 肝病儿童的选择。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关于轮状病毒在“人类胆道闭锁”中的作用的研究；在这项研究中，我们正在研究 BA 和遗传性胆汁淤积对照婴儿的血清（在 BARC 指导委员会批准的一项辅助研究中提供给我们），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) 继续参与囊性纤维化肝病 (CFLD) 的前瞻性纵向研究，旨在确定肝病发展和结果的预测因素。 4) 继续为儿科肝病研究人员提供培训机会，例如我们提议培训一名儿科胃肠病学一年级研究员进行与 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志物。 5) 继续通过出版物以及 BARC 和 CLiC 网站向科学界和非专业界提供有关儿科肝病的教育，特别注重征求使用网站的家庭对网站的反馈。 6) 继续我们已经创建的基础设施来执行 BARC、CLiC 和 CFLD。我们是致力于参与这个多中心研究小组，并相信通过该小组将有可能在改善患有这些严重小儿肝脏疾病的儿童健康的目标方面取得重大进展。