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首页> 美国卫生研究院文献>Therapeutic Advances in Chronic Disease >A review of the medical treatment of primary sclerosing cholangitis in the 21st century
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A review of the medical treatment of primary sclerosing cholangitis in the 21st century

机译：21世纪原发性硬化性胆管炎的医学研究述评

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Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease that progresses to end-stage liver disease and cirrhosis. Recurrent biliary inflammation is thought to lead to dysplasia, and as such PSC confers a high risk of cholangiocarcinoma. PSC accounts for 10% of all UK liver transplants, although transplantation does not guarantee a cure with 20% recurrence in the graft. At present there are no effective medical treatment options for PSC, and trials of novel therapeutic agents are limited by the time taken to reach clinically significant endpoints with no well defined early surrogate markers for disease outcome. Moreover, PSC appears to be a heterogeneous disease with regards to disease distribution, associated inflammatory bowel disease and subsequent disease outcome, further compounding the issue. Thus existing trials have taken place in heterogeneous groups, are likely to be underpowered to detect any individual subgroups effect. The current mainstay of medical treatment is still with ursodeoxycholic acid, although there is no evidence that it alters long-term outcome. Small pilot studies of immunosuppressive agents have taken place, but despite evidence that may support studies in larger groups, these have not been conducted. Recent advances in our understanding of the disease pathogenesis may therefore pave the way for trials of novel therapeutic agents in PSC, even given the limitations described. This review explores the controversial evidence underlying current treatment strategies and discounted treatments, and explores prospective agents that may bring new hope to the treatment of PSC in the 21st century.
机译：原发性硬化性胆管炎（PSC）是一种慢性胆汁淤积性肝病，可发展为终末期肝病和肝硬化。胆道发炎的反复发作被认为可导致不典型增生，因此PSC赋予胆管癌的高风险。 PSC占英国所有肝移植的10％，尽管移植不能保证治愈率达到20％。目前，对于PSC尚无有效的药物治疗选择，并且新型治疗药物的试验受到达到临床上显着终点所需时间的限制，而这些终点尚无明确的疾病预后替代指标。此外，就疾病分布，相关的炎症性肠病和随后的疾病结果而言，PSC似乎是一种异质性疾病，使问题更加复杂。因此，现有的试验已在异类组中进行，可能不足以检测任何单个亚组的作用。尽管没有证据表明熊去氧胆酸会改变长期结果，但目前的医学治疗仍然是乌斯脱氧胆酸。免疫抑制剂的小规模试点研究已经进行，但是尽管有证据支持较大的人群研究，但尚未进行。因此，即使有上述限制，我们对疾病发病机理的最新进展也可能为在PSC中试验新型治疗药物铺平道路。这篇综述探讨了当前治疗策略和折扣治疗的有争议证据，并探讨了可能为21世纪PSC治疗带来新希望的前瞻性药物。

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