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原发性胆汁性胆管炎的治疗药物新进展。

Emerging drugs for the treatment of primary biliary cholangitis.

机构信息

Institute of Translational and Clinical Research, Newcastle University , Newcastle-upon-Tyne, UK.

Hepatology Department, Newcastle Hospital NHS Foundation Trust , Newcastle-upon-Tyne, UK.

出版信息

Expert Opin Emerg Drugs. 2020 Jun;25(2):101-112. doi: 10.1080/14728214.2020.1751814. Epub 2020 May 5.

Abstract

INTRODUCTION

Primary biliary cholangitis (PBC) is a progressive inflammatory autoimmune cholestatic liver disease. Without treatment, it may result in fibrosis and eventually end stage liver disease. In addition to the disease burden, the symptom impact on the quality of life for PBC patients is significant. Ursodeoxycholic acid, and the second-line therapy, Obeticholic acid, are the only available licensed treatments. Although there has been rapid development of novel therapies in recent years for the treatment of PBC, there are very few symptoms directed therapies.

AREA COVERED

This literature review aims to review the current treatment landscape in PBC and to explore how the next few years may unfold in the field. The current guidelines and emerging therapies in phase 2, 3 and 4 clinical trials have been included.

EXPERT OPINION

The currently available therapies are effective, but their use has limitations and challenges and there is still significant unmet need. Although there have been promising therapeutic interventions in recent years, further research into personalizing therapeutic strategies with available treatments and new agents is needed.

摘要

简介

原发性胆汁性胆管炎(PBC)是一种进行性炎症性自身免疫性胆汁淤积性肝病。未经治疗,可能会导致纤维化,最终发展为终末期肝病。除了疾病负担外,PBC 患者的症状对生活质量的影响也很大。熊去氧胆酸和二线治疗药物奥贝胆酸是唯一获得批准的治疗药物。尽管近年来针对 PBC 的治疗方法有了快速发展,但针对症状的治疗方法却非常有限。

涵盖领域

本文献综述旨在回顾 PBC 的当前治疗现状,并探讨未来几年该领域的发展趋势。本综述纳入了目前的指南和处于 2、3、4 期临床试验阶段的新兴疗法。

专家意见

目前可用的治疗方法是有效的,但它们的使用存在局限性和挑战,仍有很大的未满足需求。尽管近年来有一些很有前景的治疗干预措施,但仍需要进一步研究如何利用现有治疗药物和新药物制定个体化的治疗策略。

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