Mazzetti Marta, Marconi Giulia, Mancinelli Martina, Benedetti Antonio, Marzioni Marco, Maroni Luca
Clinic of Gastroenterology and Hepatology, Università Politecnica delle Marche, 60126 Ancona, Italy.
Department of Gastroenterology, Azienda Sanitaria Unica Regionale Marche Area Vasta 3, 62100 Macerata, Italy.
J Clin Med. 2021 Apr 18;10(8):1763. doi: 10.3390/jcm10081763.
Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are two chronic cholestatic liver diseases affecting bile ducts that may progress to biliary cirrhosis. In the past few years, the increasing knowledge in the pathogenesis of both diseases led to a growing number of clinical trials and possible new targets for therapy. In this review, we provide an update on the treatments in clinical use and summarize the new drugs in trials for PBC and PSC patients. Farnesoid X Receptor (FXR) agonists and Pan-Peroxisome Proliferator-Activated Receptor (PPAR) agonists are the most promising agents and have shown promising results in both PBC and PSC. Fibroblast Growth Factor 19 (FGF19) analogues also showed good results, especially in PBC, while, although PBC and PSC are autoimmune diseases, immunosuppressive drugs had disappointing effects. Since the gut microbiome could have a potential role in the pathogenesis of PSC, recent research focused on molecules that could change the microbiome, with good results. The near future of the medical management of these diseases may include new treatments or a combination of multiple drugs targeting different signaling pathways at different stages of the diseases.
原发性胆汁性胆管炎（PBC）和原发性硬化性胆管炎（PSC）是两种影响胆管的慢性胆汁淤积性肝病，可进展为胆汁性肝硬化。在过去几年中，对这两种疾病发病机制的认识不断增加，导致临床试验数量不断增多，并有了可能的新治疗靶点。在本综述中，我们提供了目前临床使用的治疗方法的最新情况，并总结了针对PBC和PSC患者的试验中的新药。法尼醇X受体（FXR）激动剂和全过氧化物酶体增殖物激活受体（PPAR）激动剂是最有前景的药物，在PBC和PSC中均显示出有希望的结果。成纤维细胞生长因子19（FGF19）类似物也显示出良好的效果，尤其是在PBC中，而尽管PBC和PSC是自身免疫性疾病，但免疫抑制药物的效果却令人失望。由于肠道微生物群可能在PSC的发病机制中发挥潜在作用，最近的研究集中在能够改变微生物群的分子上，并取得了良好的结果。这些疾病的药物治疗的近期前景可能包括新的治疗方法或多种药物的联合使用，这些药物针对疾病不同阶段的不同信号通路。
