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.

虽然每种都相对罕见，但先天性胆汁淤积性肝病共同影响儿科 健康显著。其中，胆道闭锁（BA）是小儿最常见的原因， 终末期肝病和儿科肝移植的主要适应症。的 儿童肝病研究网络（ChiLDReN）成立于2009年，旨在扩大 胆道闭锁研究联盟的范围，以研究许多罕见的先天性胆汁淤积性 肝脏疾病，包括Alagille综合征、进行性家族性肝内胆汁淤积症、a1- 抗胰蛋白酶缺乏、线粒体肝病和胆盐合成缺陷。从那时起， NIDDK资助的联合体报告了许多高影响观测。 原发性硬化性胆管炎目前正在被网络添加为重点疾病。 儿童医院洛杉矶（CHLA）临床研究中心一直非常活跃， 网络的努力。在此申请中，我们重申我们的承诺，以改善 先天性肝脏疾病患者的临床结局。这一总体目标 应用将通过以下方式影响罕见肝病儿童的生存率：（1）招募 受试者进入ChiLDREN联盟内的各种临床研究和试验，以及（2） 翻译辅助研究集中在辅酶Q10作为肝纤维化的生物标志物。中央 目标#2假设是辅酶Q10的血清水平与肝纤维化的程度相关。这 该项目具有创新性，因为（1）CHLA拥有唯一的儿科首席研究员 外科医生，从而为指导委员会提供外科见解和观点，否则 CoQ 10是一种新的潜在的肝纤维化生物标志物。