Establichment of CHLA's ChilDREN Clinical Center

CHLA儿童临床中心成立

基本信息

批准号：
7743218
负责人：
KASPER SAONUN WANG
金额：
$ 21.35万
依托单位：
CHILDREN'S HOSPITAL OF LOS ANGELES
依托单位国家：
美国
项目类别：
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/7743218
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 Fibroblasts Funding Growth Health Personnel Hepatic Hospitals Individual Infant Knowledge Liver Liver diseases Los Angeles Mails Manuals Mitochondria Operative Surgical Procedures Oranges Pathogenesis Patients Population Heterogeneity Process Progressive intrahepatic cholestasis Protocols documentation Publications Rare Diseases Recruitment Activity Research Research Ethics Committees Research Infrastructure Research Personnel Safety Shipping Ships Signal Pathway Signal Transduction Site Visit Source Specimen Stem cells Steroids Testing alpha 1-Antitrypsin Deficiency base cohort experience meetings neonatal hepatitis notch protein outreach repository symposium tool

项目摘要

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）：婴儿和小儿胆汁淤积性肝病是罕见但毁灭性的疾病。 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胆汁淤积的肝财团统称儿童胆汁淤积性肝病的绝大多数原因。儿童肝病研究和教育网络（儿童）将充当合并的实体，通过该实体，这两个以前由NIH资助的网络的研究工作将继续协调关注这些严重的儿科肝病的调查工作。目前，来自洛杉矶儿童医院（CHLA）为南加州大量和种族多样化人口的患者均未招募到任何一个网络中。为了在CHLA建立新的儿童CC，我们提出以下具体目的：1）在儿童中运营CHLA儿童CC组织基础设施，筛查和入学符合条件的受试者，以收集临床数据和标本，以进行测试，处理，存储，存储和运输到财团回复库；参与胆道闭锁类固醇安全性和功效临床试验。2）建立向该地区推广的机制，包括洛杉矶县，奥兰治， Ventura，San Luis Obispo，Kern，Riverside和San Bernardino，包括免费的推荐线和将经过IRB批准的文档分发给所有婴儿胆固醇肝病病例的潜在医疗保健提供者推荐来源的网络；并开发教育工具，以提高对小儿肝病的认识，包括在北方医院进行教育巡回赛以及大规模的教育传单邮件。3）与其他CC的研究人员合作，并通过正在进行的呼吁，会议，会议，并提供候补研究的科学投入和提议，参与儿童的组织和运作；辅助项目建议：表征与肝祖细胞命运有关的信号通路的变化有助于BA和AGS的发病机理。相关性：婴儿和小儿胆汁淤积性肝病很少见。一个人，个别医院无法积累足够的经验来做出有意义的科学贡献，这是这些疾病管理算法的基础。儿童将促进较大的这些罕见疾病患者同类群的应计，以提高对这些毁灭性疾病的知识和治疗。