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原发性硬化性胆管炎的新兴药物治疗方法。

Emerging pharmacologic therapies for primary sclerosing cholangitis.

作者信息

Cheung Angela C, Lazaridis Konstantinos N, LaRusso Nicholas F, Gores Gregory J

机构信息

Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, Minnesota, USA.

出版信息

Curr Opin Gastroenterol. 2017 May;33(3):149-157. doi: 10.1097/MOG.0000000000000352.

Abstract

PURPOSE OF REVIEW

The only currently approved treatment for primary sclerosing cholangitis (PSC) is liver transplantation, with a median time to transplant of 12-18 years after diagnosis. There are a number of emerging drugs that have the potential to meet this critically unmet need that will be summarized and discussed herein.

RECENT FINDINGS

Although the cause of PSC is unknown, there are a number of novel therapeutics under development. These drugs target presumed pathogenic mechanisms largely extrapolated from ex-vivo and in-vivo preclinical models, as well as translational observations.

SUMMARY

Future therapeutic strategies for PSC may include a multitude of complex pathogenic mechanisms encompassing pathways of immunomodulation, the microbiome and inflammation-related fibrosis.

摘要

综述目的

目前唯一被批准用于原发性硬化性胆管炎(PSC)的治疗方法是肝移植,诊断后至移植的中位时间为12至18年。有多种新兴药物有潜力满足这一迫切未被满足的需求,本文将对其进行总结和讨论。

最新发现

尽管PSC的病因尚不清楚,但有多种新型治疗方法正在研发中。这些药物主要针对从体外和体内临床前模型以及转化观察中推断出的假定致病机制。

总结

PSC未来的治疗策略可能包括多种复杂的致病机制,涵盖免疫调节、微生物群和炎症相关纤维化等途径。

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本文引用的文献

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Primary Sclerosing Cholangitis.原发性硬化性胆管炎
N Engl J Med. 2016 Sep 22;375(12):1161-70. doi: 10.1056/NEJMra1506330.
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Cochrane Database Syst Rev. 2015 Dec 2;2015(12):CD011661. doi: 10.1002/14651858.CD011661.pub2.
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Sclerosing Cholangitis in Children and Adolescents.儿童和青少年硬化性胆管炎。
Clin Liver Dis. 2016 Feb;20(1):99-111. doi: 10.1016/j.cld.2015.08.008. Epub 2015 Oct 6.

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