BILLARY ATRESIA (BA) CLINICAL RESEARCH-CORTICOSTEROID THERAPY IN INFANTS WITH BA

胆道闭锁 (BA) 临床研究 - BA 婴儿的皮质类固醇治疗

• 批准号: 7380583
• 负责人: BENJAMIN L SHNEIDER
• 金额: $ 1.29万
• 依托单位: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-17 至 2007-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7380583
• 关键词: bile; clinical research; corticosteroids

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Biliary atresia is the most common cause of cholestasis (bile flow blockage) in infants and the most frequent indication for pediatric liver transplantation. The disease results from a destructive inflammatory process that affects intra- and extrahepatic bile ducts, leading to fibrosis and obliteration of the biliary tract. Although little is known about the etiology or pathogenesis of biliary atresia, epidemiologic and virologic studies point to a complex trait disorder, in which environmental factors trigger an inflammatory process that recognizes and abnormally targets the biliary system during a specific phase of postnatal development. Regardless of initiating (environmental) and modifying (genetic) factors for disease development, the inflammatory and fibrosing destruction of the biliary epithelium is common to all clinical forms of biliary atresia. Portoenterostomy (Kasai procedure utilizing the intestine to make an artificial bile duct) is the only operative procedure used currently to improve bile drainage in infants with biliary atresia. Although prompt diagnosis and surgical intervention may induce bile flow, progression to end-stage liver disease occurs in over 50% of the patients by 2 years of age. In this setting, the potential decrease of this inflammatory component by corticosteroid treatment may result in improved bile flow and better outcome after portoenterostomy. We propose a multi-center randomized, double-blinded, placebo-controlled trial to prospectively determine the efficacy of corticosteroids on the outcome of infants with biliary atresia. The trial will be conducted by the NIDDK-funded network of nine clinical centers comprising the Biliary Atresia Clinical Research Consortium (BARC), whose goal is to study the etiology, pathogenesis, diagnosis, and treatment of infants with biliary atresia. Hypothesis: In this clinical trial we propose to objectively determine whether corticosteroid treatment improves bile flow in infants with biliary atresia.

该子项目是利用 NIH/NCRR 资助的中心拨款提供的资源的众多研究子项目之一。子项目和研究者 (PI) 可能已从另一个 NIH 来源获得主要资金，因此可以在其他 CRISP 条目中得到体现。列出的机构是中心的机构，不一定是研究者的机构。胆道闭锁是婴儿胆汁淤积（胆汁流阻塞）的最常见原因，也是小儿肝移植最常见的适应症。该疾病是由影响肝内和肝外胆管的破坏性炎症过程引起的，导致胆道纤维化和闭塞。尽管对胆道闭锁的病因或发病机制知之甚少，但流行病学和病毒学研究指出这是一种复杂的特征性疾病，其中环境因素触发炎症过程，在出生后发育的特定阶段识别并异常靶向胆道系统。 无论疾病发展的起始（环境）和修饰（遗传）因素如何，胆道上皮的炎症和纤维化破坏对于所有临床形式的胆道闭锁来说都是常见的。门肠造口术（利用肠道制造人工胆管的 Kasai 手术）是目前用于改善胆道闭锁婴儿胆汁引流的唯一手术方法。尽管及时诊断和手术干预可能会引起胆汁流动，但超过 50% 的患者在 2 岁时会进展为终末期肝病。在这种情况下，通过皮质类固醇治疗可能减少这种炎症成分，可能会导致胆汁流量改善和门肠造口术后更好的结果。 我们提出了一项多中心随机、双盲、安慰剂对照试验，以前瞻性地确定皮质类固醇对胆道闭锁婴儿预后的疗效。该试验将由 NIDDK 资助的由九个临床中心组成的网络进行，该网络包括胆道闭锁临床研究联盟 (BARC)，其目标是研究胆道闭锁婴儿的病因、发病机制、诊断和治疗。 假设：在这项临床试验中，我们建议客观地确定皮质类固醇治疗是否可以改善胆道闭锁婴儿的胆汁流量。