Establichment of CHLA's ChilDREN Clinical Center

CHLA儿童临床中心成立

• 批准号: 8545822
• 负责人: KASPER SAONUN WANG
• 金额: $ 33.9万
• 依托单位: CHILDREN'S HOSPITAL OF LOS ANGELES
• 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 2014-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8545822
• 关键词: Alagille Syndrome; Algorithms; Ancillary Study; Awareness; Bile Acid Biosynthesis Pathway; Biliary Atresia; California; Child; Childhood; Clinical; Clinical Data; Clinical Trials; Collection; Consent Forms; County; Cystic Fibrosis; Defect; Disease; Education; Enrollment; Fibroblast Growth Factor; Funding; Health Personnel; Hepatic; Hospitals; Individual; Infant; Institutional Review Boards; Knowledge; Liver; Liver diseases; Los Angeles; Mails; Manuals; Mitochondria; Oranges; Pathogenesis; Patients; Population Heterogeneity; Process; Progressive intrahepatic cholestasis; Protocols documentation; Publications; Rare Diseases; Recruitment Activity; Research; Research Infrastructure; Research Personnel; Safety; Shipping; Ships; Signal Pathway; Signal Transduction; Site Visit; Source; Specimen; Stem cells; Steroids; Testing; United States National Institutes of Health; alpha 1-Antitrypsin Deficiency; base; clinical efficacy; cohort; experience; meetings; neonatal hepatitis; notch protein; operation; outreach; repository; symposium; tool

DESCRIPTION (provided by applicant): Infant and pediatric cholestatic liver diseases are rare yet devastating conditions. Biliary Atresia (BA) and idiopathic neonatal hepatitis, studied under the Biliary Atresia Research Consortium, along with Alagille syndrome (AGS), alpha-1-antitrypsin deficiency, progressive intrahepatic cholestasis, bile acid synthesis defects, mitochondrial hepatopathies, and cystic fibrosis liver disease, studied under the Cholestatic Liver Consortium collectively represent the vast majority of causes of cholestatic liver diseases in children. The Childhood Liver Disease Research and Education Network (ChiLDREN) will serve as the merged entity by which the research efforts from both previously NIH-funded networks will continue to coordinate investigative efforts focused on these significant pediatric liver diseases. Currently, patients from the large and ethnically diverse population of Southern California served by Childrens Hospital Los Angeles (CHLA) are not recruited into either network. In order to establish a new ChiLDREN CC at CHLA, we propose the following specific aims: 1) Operationalize the CHLA ChiLDREN CC organizational infrastructure, screen and enroll eligible subjects in ChiLDREN for collection of clinical data and specimens for testing, processing, storage, and shipping to the consortium repositories; participate in the biliary atresia steroid safety and efficacy clinical trial.2) Establish mechanisms for outreach into the region including the counties of Los Angeles, Orange, Ventura, San Luis Obispo, Kern, Riverside, and San Bernardino including a toll-free referral line and distribute IRB-approved documents to network of potential healthcare provider referral sources for all infant cholestatic liver disease cases; and develop educational tools to increase awareness of pediatric liver diseases including education rounds at outlying hospitals as well as mass mailings of educational flyers.3) Collaborate with the other CC investigators and participate in the organization and operation of ChiLDREN through ongoing conference calls and meetings and provision of scientific input and proposals for ancillary studies; ancillary project proposal: Characterize alterations in signaling pathways involved in hepatic progenitor cell fate contribute to the pathogenesis of BA and AGS. Relevance: Infant and pediatric cholestatic liver diseases are rare. Alone, individual hospitals cannot accumulate sufficient experience to make meaningful scientific contributions which are the basis for the management algorithms of these diseases. ChiLDREN will facilitate the accrual of larger cohorts of patients with these rare diseases to advance knowledge and treatment of these devastating diseases.

描述（由申请人提供）： 婴儿和儿童胆汁淤积性肝病是罕见但具有破坏性的疾病。胆道闭锁 (BA) 和特发性新生儿肝炎，在胆道闭锁研究联盟下进行研究，以及 Alagille 综合征 (AGS)、α-1-抗胰蛋白酶缺乏症、进行性肝内胆汁淤积、胆汁酸合成缺陷、线粒体肝病和囊性纤维化肝病，在 胆汁淤积性肝病联盟代表了儿童胆汁淤积性肝病的绝大多数病因。儿童肝病研究和教育网络 (ChiLDREN) 将作为合并后的实体，之前由 NIH 资助的两个网络的研究工作将继续协调针对这些重要儿科肝病的调查工作。目前，洛杉矶儿童医院 (CHLA) 所服务的南加州人口众多且种族多样的患者并未被招募到这两个网络中。为了在 CHLA 建立一个新的 ChiLDREN CC，我们提出以下具体目标： 1) 运行 CHLA ChiLDREN CC 组织基础设施，在 ChiLDREN 中筛选和注册符合条件的受试者，以收集临床数据和样本进行测试、处理、存储和运送到联盟存储库；参与胆道闭锁类固醇安全性和有效性临床试验。2) 建立向洛杉矶县、奥兰治县、 文图拉、圣路易斯奥比斯波、克恩、里弗赛德和圣贝纳迪诺，包括免费转介热线和 将 IRB 批准的文件分发给所有婴儿胆汁淤积性肝病病例的潜在医疗保健提供者转介来源网络；开发教育工具以提高对儿科肝病的认识，包括在边远医院进行教育查房以及大量邮寄教育传单。3) 与其他 CC 研究者合作，通过持续的电话会议和会议以及为辅助研究提供科学投入和建议，参与 ChiLDREN 的组织和运作；辅助项目提案：表征参与肝祖细胞命运的信号通路的改变有助于 BA 和 AGS 的发病机制。 相关性：婴儿和儿童胆汁淤积性肝病很少见。仅靠个别医院无法积累足够的经验来做出有意义的科学贡献，而这些贡献是这些疾病的管理算法的基础。 ChiLDREN 将促进更多患有这些罕见疾病的患者群体的增加，以增进对这些破坏性疾病的了解和治疗。