Limited Competition for the Continuation of the Childhood Liver Disease Research Network (ChilLDRen) Clinical Centers.

儿童肝病研究网络 (ChilLDRen) 临床中心延续的有限竞争。

• 批准号: 10019514
• 负责人: KASPER SAONUN WANG
• 金额: $ 42.34万
• 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 2024-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10019514
• 关键词: Address; Alagille Syndrome; Algorithms; Ancillary Study; Biliary Atresia; Biological Markers; Child; Childhood; Clinical; Clinical Research; Clinical Trials; Coenzyme Q10; Data; Defect; Diagnosis; Disease; Enrollment; Fibrosis; Funding; Goals; Health; Histologic; Hospitals; Human; Individual; Infant; Ligation; Liver Dysfunction; Liver Fibrosis; Liver diseases; Logic; Los Angeles; Measurement; Mus; National Institute of Diabetes and Digestive and Kidney Diseases; Operative Surgical Procedures; Patient-Focused Outcomes; Patients; Pediatric Hospitals; Plasma; Principal Investigator; Progressive intrahepatic cholestasis; Rare Diseases; Reporting; Research; Research Proposals; Sampling; Serum; Surgeon; Validation; bile duct; bile salts; cholestatic liver disease; chronic liver disease; clinical center; clinical research site; cohort; experience; improved; innovation; insight; liver transplantation; novel; novel marker; point of care; potential biomarker; primary sclerosing cholangitis; success

Albeit each is relatively rare, congenital cholestatic liver diseases collectively impact pediatric health significantly. Amongst them, biliary atresia (BA) is the most common cause of pediatric end-stage liver disease and the leading indication for pediatric liver transplantation. The Childhood Liver Disease Research Network (ChiLDReN) was established in 2009 to expand the scope of the Biliary Atresia Research Consortium to study numerous rare congenital cholestatic liver diseases including Alagille syndrome, Progressive Familial Intrahepatic Cholestasis, a1- Antitrypsin Deficiency, Mitochodrial Hepatopathies, and Bile Salt Synthesis Defects. Since then, numerous high impact observations have been reported by the NIDDK-funded consortium. Primary Sclerosing Cholangitis is currently being added as a disease of focus by the network. The Children’s Hospital Los Angeles (CHLA) clinical research center has been very active in all aspects of the network’s efforts. In this application, we restate our commitment to improve the clinical outcomes of patients with congenital liver diseases. The overall objectives of this application are to impact survival of children with rare liver diseases via (1) the enrollment of subjects into the various clinical studies and trials within the ChiLDREN consortium and (2) a translational ancillary study focused on CoQ10, as a biomarker of liver fibrosis. The central hypothesis of objective #2 is that serum levels of CoQ10 correlate with extent of liver fibrosis. This project is innovative because (1) CHLA has the only Principal Investigator who is a pediatric surgeon, thus providing surgical insight and perspective for a Steering Committee otherwise comprised of pediatric hepatologists and (2) CoQ10 is a novel potential biomarker for liver fibrosis.

尽管每个人都相对罕见，但先天性胆固醇肝疾病统称为儿科 健康。其中，胆道闭锁（BA）是小儿最常见的原因 末期肝病和小儿肝移植的主要指示。这 儿童肝病研究网络（儿童）于2009年建立，以扩大 胆道闭锁研究联盟的范围，研究了许多罕见的先天性胆汁淤积 肝病，包括阿拉吉综合征，进行性家族性肝内胆汁淤积，A1- 抗胰蛋白酶缺乏症，线路肝病和胆汁盐合成缺陷。自那以后， NIDDK资助的财团报告了许多高影响观察结果。 该网络目前正在将原发性硬化性胆管炎作为重点疾病添加。 儿童医院洛杉矶（CHLA）临床研究中心非常活跃 网络努力的各个方面。在此应用中，我们重述了我们的承诺 先天性肝病患者的临床结局。总体目标 应用是通过（1）入学 受试者进入各种临床研究和儿童财团内的试验，（2）a 翻译辅助研究的重点是COQ10，作为肝纤维化的生物标志物。中央 目标＃2的假设是COQ10的血清水平与肝纤维化程度相关。这 项目具有创新性，因为（1）CHLA是唯一的主要研究人员 外科医生，从而为指导委员会提供手术见解和观点 小儿肝病学家和（2）COQ10是肝纤维化的新型潜在生物标志物。