Clinical Research on Nonalcoholic Fatty Liver Disease

项目摘要/摘要 哥伦比亚大学非酒精性脂肪性肝炎临床研究网络(NASH CRN) 它在加州大学圣地亚哥分校的分包网站发挥了重要作用 近16年来加深对儿童非酒精性脂肪性肝病的认识 和成年人。鉴于NAFLD是美国最常见的慢性肝病， 已知的后果包括肝硬变、肝移植和肝癌，我们必须 制定更好的非侵入性措施，以诊断更严重或进行性形式的 NAFLD，我们找到了具有独特和合理靶点的改进疗法。其中的建议是 我们临床中心有限的竞争更新展示了在纵向上取得的进步 数据库研究和过去和当前/正在进行的随机临床试验中的重要发现 成人和儿童。我们的中心和网络描述了完成和继续的计划 临床试验的发展，寻找新的生物成像方法来评估对 治疗，定义和评估成像作为一种非侵入性标记的性能， 可用作NAFLD/NASH某些功能的代理，并可用作 跟踪疾病进展并完成和开发辅助研究以了解 遗传和环境因素在调节疾病过程中的作用。在这方面， 我们领导研究开发蛋白质组学试剂作为预测和诊断标记物 在阐明肠道微生物组的作用、核激素受体的作用以及 遗传变异在儿童非酒精性脂肪肝中的作用。我们打算继续在 NASH CRN，作为PI是指导委员会和数据库委员会的联合主席 执行委员会主席兼儿科委员会主席。我们中心在以下方面的主导作用 招生，以及我们在招募和留住成人、儿童和少数族裔方面的领导 在NASH，CRN预计将继续，直到完成目前的试验和启动 新提出的研究。

Project Summary/Abstract The Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) at Columbia University and its subcontracted sites at the University of California, San Diego have played a major role over the past 16 years in furthering the understanding of nonalcoholic fatty liver disease in children and adults. Given that NAFLD is the most common chronic liver disease in the United States and that known outcomes include cirrhosis, liver transplant and liver cancer, it is imperative that we develop better noninvasive measures to diagnose with more severe or progressive forms of NAFLD and that we find improved therapies with unique and rational targets. The proposal in this limited competition renewal for our Clinical Center demonstrates the progress made in longitudinal database studies and significant findings in past and current/ongoing randomized clinical trials in adults and children. Our Center and the Network describe plans for completion and continued development of clinical trials, for finding novel bio-imaging methods to assess response to therapy, to define and evaluate the performance of imaging as a noninvasive marker that might be used as a surrogate for certain features of NAFLD/NASH and as a means of tracking disease progression and to complete and develop ancillary studies for understanding the role of genetic and environmental factors in modulating the course of disease. In this regard, we lead studies in developing proteomic reagents as predictive and diagnostic markers for NAFLD; in elucidating the role of the gut microbiome, the role of nuclear hormone receptors, and the role of genetic variants in pediatric NAFLD. We intend to continue to play leading roles in the NASH CRN, as the PI is Co-Chair of the Steering Committee and Database Committee, a member of the Executive Committee, and Chair of the Pediatric Committee. Our Centers’ leading role in enrollment, along with our leadership in recruitment and retention of adults, children and minorities in the NASH CRN are expected to continue through completion of current trials and initiation of newly proposed studies.