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Emerging drugs for the treatment of primary biliary cholangitis.
Expert Opinion on Emerging Drugs ( IF 3.4 ) Pub Date : 2020-05-05 , DOI: 10.1080/14728214.2020.1751814
Naw April Phaw 1, 2 , Jessica Katharine Dyson 1, 2 , David Jones 1, 2, 3
Affiliation  

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.



中文翻译:

用于治疗原发性胆源性胆管炎的新兴药物。

介绍

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。如果不进行治疗,可能会导致纤维化,最终导致晚期肝病。除疾病负担外,症状对PBC患者的生活质量影响很大。熊去氧胆酸和二线治疗奥贝胆酸是唯一可用的许可治疗。尽管近年来用于PBC治疗的新型疗法发展迅速,但很少有针对症状的疗法。

覆盖面积

这篇文献综述旨在回顾PBC目前的治疗前景,并探讨该领域未来几年的发展趋势。目前的指南和2、3和4期临床试验中出现的新疗法已包括在内。

专家意见

当前可用的疗法是有效的,但是它们的使用具有局限性和挑战,并且仍然存在大量未满足的需求。尽管近年来有前景可观的治疗干预措施,但需要进一步研究以可用的治疗方法和新药物个性化治疗策略。

更新日期:2020-07-02
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