The Mount Sinai Biliary Atresia Study Group

西奈山胆道闭锁研究小组

• 批准号: 6668539
• 负责人: BENJAMIN L SHNEIDER
• 金额: $ 16.85万
• 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 2007-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6668539
• 关键词: beta antiadrenergic agent; biliary atresia; blood banks; chemotherapy; child (0-11); cholestasis; clinical research; clinical trials; cooperative study; endoscopy; human therapy evaluation; infant human (0-1 year); information systems; liver transplantation; medical complication; patient oriented research; portal hypertension; propranolol; tissue resource /registry

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证。尽管胆道闭锁具有重要的临床意义，但人们对胆道闭锁的各种基本问题知之甚少。病毒和遗传因素引起的胆道闭锁已经被探索，然而其病因仍然从根本上未知。由于缺乏对胆道闭锁儿童的前瞻性临床随访，导致对其自然病史的了解相对较差。胆道闭锁的内科和外科治疗缺乏循证方法，这在很大程度上是由于缺乏前瞻性的多中心研究。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的假设，即一个国际多中心胆道闭锁研究联盟的支持将允许对胆道闭锁的病因、自然病史和治疗进行有效的研究。西奈山胆道闭锁研究小组将由设在纽约、新泽西、康涅狄格和波多黎各的12个中心组成的现有临床联盟组成。本研究小组为胆道闭锁临床研究联盟提出了以下三个具体目标： 目的1：建立胆道闭锁数据库和血清/组织库：前瞻性收集新生儿胆汁淤积症患儿的临床数据库和血清及组织库，对提高我们对胆道闭锁的认识具有绝对重要的意义。 目的2.对未行肝门肠吻合术或肝门肠吻合术失败的胆道闭锁患儿进行前瞻性分析，以确定失败的原因并确定最佳的肝移植入路。 目的3.β-受体阻滞剂预防EHBA患儿门脉高压并发症的随机试验6-9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，并前瞻性评估门脉高压并发症。