A RANDOMIZED, DOUBLE-BLINDED, PLACEBO-CONTROLLED TRIAL OF CORTICOSTEROID THERAPY

皮质类固醇治疗的随机、双盲、安慰剂对照试验

基本信息

  • 批准号:
    8356692
  • 负责人:
  • 金额:
    $ 0.67万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2010
  • 资助国家:
    美国
  • 起止时间:
    2010-12-01 至 2011-11-30
  • 项目状态:
    已结题

项目摘要

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. ABSTRACT Biliary atresia is the most common cause of cholestasis 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. Based on these data, the following unifying pathogenesis model can be preliminarily proposed: 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. In this setting, the potential decrease of this inflammatory component by corticosteroid treatment may result in improved bile flow and better outcome after portoenterostomy. Therefore, in this clinical trial we propose to objectively determine whether corticosteroid treatment improves bile flow in infants with biliary atresia. The significance of the proposed trial is that it will determine whether corticosteroids are an effective medical treatment to improve bile drainage and long-term outcome, and whether its use reduces the need for liver transplantation in infants with biliary atresia. The trial will be performed by the NIH-supported Biliary Atresia Research Consortium (BARC). BARC has the infrastructure to prospectively follow a sufficiently large number of patients and to collect samples necessary for clinical research studies addressing etiology, pathogenesis, diagnosis, and treatment of children with biliary atresia.
这个子项目是许多利用资源的研究子项目之一 由NIH/NCRR资助的中心拨款提供。子项目的主要支持 子项目的主要研究者可能是由其他来源提供的, 包括其他NIH来源。 列出的子项目总成本可能 代表子项目使用的中心基础设施的估计数量, 而不是由NCRR赠款提供给子项目或子项目工作人员的直接资金。 摘要 胆道闭锁是婴儿胆汁淤积最常见的原因,也是小儿肝移植最常见的适应症。这种疾病是由破坏性的炎症过程引起的,影响肝内和肝外胆管,导致胆道纤维化和闭塞。虽然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了一种复杂的性状障碍,其中环境因素触发炎症过程,该过程在出生后发育的特定阶段识别并异常靶向胆道系统。基于这些数据,可以初步提出以下统一的发病机理模型: 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在所有临床形式的胆道闭锁中是常见的。在这种情况下,皮质类固醇治疗可能减少这种炎症成分,可能会导致改善胆汁流量和更好的结果后,门肠吻合。因此,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能改善胆道闭锁婴儿的胆汁流量。这项试验的意义在于,它将确定皮质类固醇是否是一种有效的药物治疗,以改善胆汁引流和长期结果,以及它的使用是否减少了胆道闭锁婴儿肝移植的需要。 该试验将由NIH支持的胆道闭锁研究联盟(BARC)进行。BARC的基础设施可以前瞻性地随访足够多的患者,并收集临床研究所需的样本,这些研究涉及胆道闭锁儿童的病因、发病机制、诊断和治疗。

项目成果

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SAUL J. KARPEN其他文献

SAUL J. KARPEN的其他文献

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{{ truncateString('SAUL J. KARPEN', 18)}}的其他基金

Modeling genetic contributions to biliary atresia
模拟遗传对胆道闭锁的影响
  • 批准号:
    10639240
  • 财政年份:
    2023
  • 资助金额:
    $ 0.67万
  • 项目类别:
Research Training in Translational Gastroenterology and Hepatology
转化胃肠病学和肝病学研究培训
  • 批准号:
    10410926
  • 财政年份:
    2016
  • 资助金额:
    $ 0.67万
  • 项目类别:
Research Training in Translational Gastroenterology and Hepatology
转化胃肠病学和肝病学研究培训
  • 批准号:
    9073070
  • 财政年份:
    2016
  • 资助金额:
    $ 0.67万
  • 项目类别:
Research Training in Translational Gastroenterology and Hepatology
转化胃肠病学和肝病学研究培训
  • 批准号:
    9280922
  • 财政年份:
    2016
  • 资助金额:
    $ 0.67万
  • 项目类别:
The Childhood Liver Disease Research and Education Network (ChilDREN)
儿童肝病研究和教育网络 (ChilDREN)
  • 批准号:
    8011891
  • 财政年份:
    2010
  • 资助金额:
    $ 0.67万
  • 项目类别:
CHOLESTATIC LIVER DISEASE CONSORTIUM (CLIC): LONGITUDINAL STUDY OF GENETIC CAUSE
胆汁淤积性肝病联盟 (CLIC):遗传原因的纵向研究
  • 批准号:
    8356694
  • 财政年份:
    2010
  • 资助金额:
    $ 0.67万
  • 项目类别:
BARC: BILLIARY ATRESIA STUDY IN INFANTS AND CHILDREN (BASIC)
BARC:婴儿和儿童胆道闭锁研究(基础)
  • 批准号:
    8356678
  • 财政年份:
    2010
  • 资助金额:
    $ 0.67万
  • 项目类别:
BILIARY ATRESIA RESEARCH CONSORTIUM (BARC): A PROSPECTIVE DATABASE OF INFANT
胆道闭锁研究联盟 (BARC):婴儿前瞻性数据库
  • 批准号:
    8356666
  • 财政年份:
    2010
  • 资助金额:
    $ 0.67万
  • 项目类别:
A RANDOMIZED, DOUBLE-BLINDED, PLACEBO-CONTROLLED TRIAL OF CORTICOSTEROID THERAPY
皮质类固醇治疗的随机、双盲、安慰剂对照试验
  • 批准号:
    8166708
  • 财政年份:
    2009
  • 资助金额:
    $ 0.67万
  • 项目类别:
CHOLESTATIC LIVER DISEASE CONSORTIUM (CLIC): LONGITUDINAL STUDY OF GENETIC CAUSE
胆汁淤积性肝病联盟 (CLIC):遗传原因的纵向研究
  • 批准号:
    8166711
  • 财政年份:
    2009
  • 资助金额:
    $ 0.67万
  • 项目类别:
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