The Mount Sinai Biliary Atresia Study Group

西奈山胆道闭锁研究小组

• 批准号: 7456349
• 负责人: FREDERICK J SUCHY
• 金额: $ 18.55万
• 依托单位: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-09-30 至 2009-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7456349
• 关键词: Adrenergic beta-Antagonists; Age-Months; Ascites; Bile fluid; Biliary Atresia; Biology; Child; Childhood; Cholestasis; Clinical; Clinical Research; Connecticut; Data; Databases; Development; Enterostomy procedure; Etiology; Failure; Health Services Accessibility; Hemorrhage; Infant; International; Lead; Liver; Liver diseases; Measures; Medical; Morbidity - disease rate; Natural History; Neonatal; New Jersey; New York; Numbers; Operative Surgical Procedures; Outcome; Patients; Placebos; Portal Hypertension; Prevention; Procedures; Puerto Rico; Randomized; Randomized Controlled Clinical Trials; Range; Relative (related person); Research; Serum; Surgical Management; Time; Tissue Banks; Tissues; Transplantation; Urine; base; bile duct; concept; follow-up; hemodynamics; insight; liver transplantation; mortality; programs; prospective; virus genetics

DESCRIPTION (provided by applicant): Biliary atresia is the leading cause of morbidity and mortality from pediatric liver disease and it is the most common indication for pediatric liver transplantation. Despite its clinical importance, little is known of a wide range of fundamental issues regarding biliary atresia. Viral and genetic causes of biliary atresia have been explored, yet its etiology remains fundamentally unknown. A lack of prospective clinical follow-up of children with biliary atresia has resulted in a relative poor understanding of its natural history. Evidence based approaches to the medical and surgical management of biliary atresia do not exist, in large part due to an absence of prospective multi-centered studies. The Biliary Atresia Clinical Research Consortium is an outstanding mechanism for advancing the study of biliary atresia. Its concept is based upon the hypothesis that support of an international multi-centered research consortium of biliary atresia will permit valid studies of the etiology, natural history and treatment of biliary atresia. The Mount Sinai Biliary Atresia Study Group will consist of a pre-existing clinical consortium of twelve centers located in New York, New Jersey, Connecticut and Puerto Rico. This study group proposes the following three specific aims for the Biliary Atresia Clinical Research Consortium: Aim 1: Establishment of a biliary atresia database and serum/tissue bank:A prospectively collected clinical database and bank of serum and tissues from children with neonatal cholestasis is of absolute critical importance to advancing our understanding of biliary atresia. Aim 2. Prospective analysis of children with biliary atresia without a hepatoportoenterostomy or with a failed hepatoportoenterostomy:Prospective analysis of these patients will be performed in order to determine the reasons for these failures and to determine the optimal approach to their liver transplantation. Aim 3. Randomized trial of beta-blockade in the prevention of complications of portal hypertension in children with EHBA.Infants between 6 and 9 months of age will be randomized to receive beta-blocker therapy or placebo and complications of portal hypertension will be assessed prospectively.

描述（由申请人提供）： 胆道闭锁是儿科肝病发病率和死亡率的主要原因，也是儿科肝移植最常见的适应症。尽管它的临床重要性，很少有人知道广泛的基本问题，关于胆道闭锁。胆道闭锁的病毒和遗传原因已被探索，但其病因仍然是根本未知的。由于缺乏对胆道闭锁儿童的前瞻性临床随访，导致对其自然史的了解相对较少。胆道闭锁的内科和外科治疗的循证方法并不存在，这在很大程度上是由于缺乏前瞻性多中心研究。胆道闭锁临床研究联盟是推进胆道闭锁研究的杰出机构。它的概念是基于这样一个假设，即支持一个国际多中心的胆道闭锁研究联盟将允许有效的研究胆道闭锁的病因，自然史和治疗。西奈山胆道闭锁研究组将由位于纽约、新泽西、康涅狄格州和波多黎各的12个中心组成的既有临床联盟组成。本研究组为胆道闭锁临床研究联盟提出了以下三个具体目标： 目标1：胆道闭锁数据库和血清/组织库的建立：前瞻性收集的新生儿胆汁淤积症患儿的临床数据库和血清和组织库对于促进我们对胆道闭锁的理解至关重要。 目标二。未行肝门肠吻合术或肝门肠吻合术失败的胆道闭锁儿童的前瞻性分析：将对这些患者进行前瞻性分析，以确定这些失败的原因，并确定肝移植的最佳方法。 目标3。β受体阻滞剂预防EHBA患儿门静脉高压并发症的随机试验。6至9个月大的婴儿将随机接受β受体阻滞剂治疗或安慰剂治疗，并前瞻性评估门静脉高压并发症。