Primary sclerosing cholangitis: what is new in the therapeutic landscape

IF 5.8 2区医学 Q1 IMMUNOLOGY

Current Opinion in Immunology Pub Date : 2025-07-18 DOI:10.1016/j.coi.2025.102613

Armando Curto , Laura Cristoferi , Marco Carbone , Andrea Galli , Pietro Invernizzi

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引用次数: 0

Abstract

Primary sclerosing cholangitis (PSC) is a rare, chronic cholestatic liver disease affecting the intra- and extra-hepatic bile ducts, leading to progressive inflammation and fibrosis. Its pathogenesis is complex and involves genetic predisposition, environmental triggers, and their interaction through the gut–liver axis, mediated by the microbiota. To date, no approved therapies modify the natural history of PSC, and liver transplantation remains the only curative option.

This review provides an overview of current investigational therapies for PSC, emphasizing their mechanisms of action and relevance to the underlying disease biology. It also examines key challenges in therapeutic development, including the lack of validated surrogate end points, clinical heterogeneity, and the confounding effects of concomitant inflammatory bowel disease treatments factors that complicate trial design and interpretation.

Clarifying these aspects is essential to support the development of effective, targeted therapies for this complex and currently untreatable condition.

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原发性硬化性胆管炎：治疗领域的新进展

原发性硬化性胆管炎（PSC）是一种罕见的慢性胆汁淤积性肝病，影响肝内和肝外胆管，导致进行性炎症和纤维化。其发病机制是复杂的，涉及遗传易感性、环境触发因素及其通过肠道-肝脏轴的相互作用，由微生物群介导。到目前为止，还没有批准的治疗方法可以改变PSC的自然史，肝移植仍然是唯一的治疗选择。本文综述了目前PSC的研究治疗方法，强调了它们的作用机制和与潜在疾病生物学的相关性。它还研究了治疗发展中的关键挑战，包括缺乏有效的替代终点，临床异质性，以及合并炎症性肠病治疗因素的混杂效应，这些因素使试验设计和解释复杂化。澄清这些方面对于支持针对这种复杂且目前无法治疗的疾病开发有效的靶向治疗方法至关重要。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Current Opinion in Immunology 医学-免疫学

CiteScore

13.30

自引率

1.40%

发文量

审稿时长

67 days

期刊介绍： Current Opinion in Immunology aims to stimulate scientifically grounded, interdisciplinary, multi-scale debate and exchange of ideas. It contains polished, concise and timely reviews and opinions, with particular emphasis on those articles published in the past two years. In addition to describing recent trends, the authors are encouraged to give their subjective opinion of the topics discussed. In Current Opinion in Immunology we help the reader by providing in a systematic manner: 1. The views of experts on current advances in their field in a clear and readable form. 2. Evaluations of the most interesting papers, annotated by experts, from the great wealth of original publications. Current Opinion in Immunology will serve as an invaluable source of information for researchers, lecturers, teachers, professionals, policy makers and students. Current Opinion in Immunology builds on Elsevier''s reputation for excellence in scientific publishing and long-standing commitment to communicating reproducible biomedical research targeted at improving human health. It is a companion to the new Gold Open Access journal Current Research in Immunology and is part of the Current Opinion and Research(CO+RE) suite of journals. All CO+RE journals leverage the Current Opinion legacy-of editorial excellence, high-impact, and global reach-to ensure they are a widely read resource that is integral to scientists'' workflow.