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原发性胆汁性胆管炎的药物治疗进展

Update on the Pharmacological Treatment of Primary Biliary Cholangitis.

作者信息

Floreani Annarosa, Gabbia Daniela, De Martin Sara

机构信息

Department of Surgery, Oncology and Gastroenterology, University of Padova, 35131 Padova, Italy.

IRCCS Negrar, 37024 Verona, Italy.

出版信息

Biomedicines. 2022 Aug 20;10(8):2033. doi: 10.3390/biomedicines10082033.

Abstract

Ursodeoxycholic acid (UDCA) is the first-line therapy used for the treatment of PBC. In recent years, new pharmacological agents have been proposed for PBC therapy to cure UDCA-non-responders. Obeticholic acid (OCA) is registered in many countries for PBC, and fibrates also seem to be effective in ameliorating biochemistry alteration and symptoms typical of PBC. Moreover, a variety of new agents, acting with different mechanisms of action, are under clinical evaluation for PBC treatment, including PPAR agonists, anti-NOX agents, immunomodulators, and mesenchymal stem cell transplantation. Since an insufficient amount of data is currently available about the effect of these novel approaches on robust clinical endpoints, such as transplant-free survival, their clinical approval needs to be supported by the consistent improvement of these parameters. The intensive research in this field will hopefully lead to a novel treatment landscape for PBC in the near future, with innovative therapies based on the combination of multiple agents acting on different pathogenetic mechanisms.

摘要

熊去氧胆酸(UDCA)是用于治疗原发性胆汁性胆管炎(PBC)的一线疗法。近年来,已提出用于PBC治疗的新药,以治愈对UDCA无反应者。奥贝胆酸(OCA)已在许多国家注册用于PBC治疗,贝特类药物似乎也能有效改善PBC典型的生化改变和症状。此外,多种作用机制不同的新药正在进行PBC治疗的临床评估,包括过氧化物酶体增殖物激活受体(PPAR)激动剂、抗NADPH氧化酶(NOX)剂、免疫调节剂和间充质干细胞移植。由于目前关于这些新方法对无移植生存期等可靠临床终点的影响的数据不足,它们的临床批准需要这些参数的持续改善来支持。该领域的深入研究有望在不久的将来为PBC带来新的治疗局面,即基于作用于不同致病机制的多种药物联合的创新疗法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fa0f/9405864/2f5cf1086b67/biomedicines-10-02033-g001.jpg

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