Childhood Liver Disease Research and Education Network Clinical Center in Seattle

西雅图儿童肝病研究和教育网络临床中心

• 批准号: 7743648
• 负责人: KAREN F MURRAY
• 金额: $ 35.78万
• 依托单位: SEATTLE CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-10 至 2014-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7743648
• 关键词: Adrenal Cortex Hormones; Alaska; Alaska Native; Allogenic; American Indians; Biliary Atresia; Biochemical Genetics; Caring; Child; Child Care; Childhood; Chronic; Clinical; Clinical Research; Coupled; Cystic Fibrosis; Databases; Diagnosis; Disease; Education; Enrollment; Etiology; Feasibility Studies; Funding; Future; Gastroenterology; Genetic; Hawaii; Hepatobiliary; Hepatology; Idaho; Immune response; Laboratory Research; Liver; Liver diseases; Longitudinal Studies; Medical; Metabolic Diseases; Microchimerism; Montana; National Institute of Diabetes and Digestive and Kidney Diseases; Natural History; Neonatal; Only Child; Operative Surgical Procedures; Oregon; Pacific Northwest; Patient Care; Patients; Pediatric Hospitals; Peripheral; Pilot Projects; Population; Positioning Attribute; Recruitment Activity; Research; Research Personnel; Research Training; Resources; Site; Training; Training Programs; United States; Universities; Washington; Work; Wyoming; experience; liver transplantation; next generation; patient population; programs; prospective; treatment trial

DESCRIPTION (provided by applicant): Biliary atresia and the other cholestatic liver diseases of childhood are important causes of pediatric liver disease. Encouraged by the significance of the research of the current BARC and CLiC consortia, the NIDDK is creating ChiLDREN, a new consortium that will replace BARC and CLiC and expand the number of research sites. Dr. Murray and her research team propose to establish a Clinical Center in Seattle as part of ChiLDREN. This Clinical Center brings together the patients of the Northwest and Alaska, investigators experienced in pediatric liver disease research and the BARC trials, the diagnosis and management of childhood cholestatic diseases, the only pediatric liver transplant center in the Northwest, and the resources of Seattle Children's and the University of Washington. The scope of the patient population, coupled with the richness of the clinical programs and experience of the investigators of this Seattle Clinical Center; assure that this research team will successfully enroll a large number of children with biliary atresia and other cholestatic liver diseases into the network's longitudinal natural history and database studies, the biliary atresia corticosteroid treatment trial, and future ancillary and pilot studies. The ability to train the next generation of pediatric liver disease researchers and contribute to the scientific understanding of the diseases via the clinical and laboratory research expertise relevant to biliary atresia at Seattle Children's, assure the contributions by the Clinical Center to forwarding the understanding of these conditions. Dr. Murray plans to establish the ChiLDREN Clinical Center in Seattle, and continue the currently approved longitudinal and treatment studies of the current BARC and CLiC Networks (Specific Aim 1). She has included a proposal for a pilot/feasibility study (Specific Aim 2) evaluating maternal microchimerism in biliary atresia and the resultant allogeneic peripheral immune response as a possible pathogenic etiology underlying this condition. Dr. Murray has also proposed training opportunities unique to the Seattle Clinical Center to help educate and train the pediatric liver disease researchers of the future (Specific Aim 3). Relevance: Biliary atresia and the other childhood cholestatic liver diseases are important causes of chronic liver disease in children, and the leading cause for liver transplantation. The initial funding period for the two relevant clinical research networks has been successful, however, expansion of the work is necessary. This next funding period will expand the currently ongoing research of natural history, genetics, and treatment, and explore answers to new questions relevant to these conditions.

描述（由申请人提供）： 胆道闭锁和其他儿童期胆汁淤积性肝病是小儿肝病的重要原因。在当前BARC和CLIC联盟的研究的重要性的鼓励下，NIDDK正在创建儿童，这是一个新的财团，将取代BARC和CLIC并扩大研究地点的数量。 Murray博士和她的研究团队建议在西雅图建立一个临床中心，作为儿童的一部分。这个临床中心汇集了西北和阿拉斯加的患者，在儿科肝病研究和BARC试验中经历的研究者，儿童胆汁淤积性疾病的诊断和管理，西北唯一的儿科肝移植中心以及西雅图儿童和华盛顿大学的资源。患者人群的范围，再加上该西雅图临床中心的临床计划和研究人员的丰富性；确保该研究团队将成功地将大量患有胆道闭锁的儿童和其他胆汁淤积性肝脏疾病纳入该网络的纵向自然史和数据库研究，胆道性胸围皮质类固醇治疗试验以及未来的辅助和试验研究。培训下一代小儿肝病研究人员的能力，并通过与西雅图儿童的临床和实验室研究专业知识有助于对疾病的科学理解，并确保临床中心的贡献，以转发对这些疾病的理解。 Murray博士计划在西雅图建立儿童临床中心，并继续对当前BARC和CLIC网络的纵向和治疗研究（具体AIM 1）。她提出了一项试验/可行性研究的建议（特定目的2）评估胆道闭锁中的母体微chimerism和由此产生的同种异体外周免疫反应，这是这种情况下的可能的致病学病因。默里（Murray）博士还提出了西雅图临床中心独有的培训机会，以帮助教育和培训未来的儿科肝病研究人员（特定目标3）。 相关性：胆道闭锁和其他儿童胆汁淤积性肝病是儿童慢性肝病的重要原因，也是肝移植的主要原因。两个相关临床研究网络的初始资金期限已成功，但是，这项工作的扩展是必要的。下一个资金时期将扩大当前正在进行的对自然史，遗传学和治疗的研究，并探索与这些条件相关的新问题的答案。