Establichment of CHLA's ChilDREN Clinical Center

CHLA儿童临床中心成立

• 批准号: 8128631
• 负责人: KASPER SAONUN WANG
• 金额: $ 30.68万
• 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 2014-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8128631
• 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的，但破坏性的条件。胆道闭锁研究联盟研究的胆道闭锁（BA）和特发性新生儿肝炎，沿着胆汁淤积性肝病联盟研究的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共同代表了儿童胆汁淤积性肝病的绝大多数原因。儿童肝脏疾病研究和教育网络（ChiLDREN）将作为合并后的实体，通过该实体，先前NIH资助的两个网络的研究工作将继续协调集中在这些重要的儿科肝脏疾病上的调查工作。目前，来自洛杉矶儿童医院（CHLA）服务的南加州庞大且种族多样化的人群的患者没有被招募到任何一个网络中。为了在CHLA建立一个新的ChiLDREN CC，我们提出了以下具体目标：1）运行CHLA ChiLDREN CC的组织基础设施，筛选和招募符合条件的受试者，以收集临床数据和标本，用于测试，处理，储存和运送到联盟储存库;参与胆道闭锁类固醇安全性和有效性临床试验。2）建立推广到该地区的机制，包括洛杉矶，橙子， 文图拉、圣路易斯奥比斯波、克恩、滨江和圣贝纳迪诺，包括免费转诊热线， 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诊来源网络;并开发教育工具，以提高对儿科肝病的认识，包括在外围医院进行教育巡回以及大规模邮寄教育传单。3）与其他CC研究者合作，通过持续的电话会议和会议参与ChiLDREN的组织和运营并为辅助研究提供科学投入和建议;辅助项目建议：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是罕见的。单个医院无法积累足够的经验，做出有意义的科学贡献，而这些贡献是这些疾病管理算法的基础。ChiLDREN将促进这些罕见疾病患者的更大队列的增加，以促进这些毁灭性疾病的知识和治疗。