难治性原发性胆汁性胆管炎的早期评估与治疗进展

Advances in the early evaluation and treatment of refractory primary biliary cholangitis

原发性胆汁性胆管炎(primary biliary cholangitis,PBC)是以胆管上皮损伤、肝内胆汁淤积为特点的慢性自身免疫性肝病,熊去氧胆酸(ursodeoxycholic acid,UDCA)是目前治疗PBC的一线药物。然而,临床约40%的PBC患者对UDCA应答不佳,称为难治性PBC,因此,尽早判断UDCA治疗效果是临床亟待解决的问题。对于此类患者,应及早进行风险评估,并在UDCA治疗的基础上联合使用二线治疗药物,从而达到治疗目标。近年来,随着对PBC发病机制的进一步阐明,以及对胆汁酸代谢和免疫功能紊乱等靶向药物的研究,从而指导临床为难治性PBC患者制定合理的治疗方案。

Primary biliary cholangitis(PBC)is an immune-mediated chronic liver disease characterized by biliary epithelial injury and intrahepatic cholestasis.And ursodeoxycholic acid(UDCA)is the first-line therapeutic drug for PBC patients.However,up to 40%of patients with PBC have an incomplete response to the first-line therapy called refractory PBC,which is an urgent clinical problem to evaluate the therapeutic effects of UDCA as soon as possible.For these patients,it is necessary to have early risk stratifications and additional treatments to reach therapeutic goals.Recently,as the mechanisms underlying the pathogenesis of PBC are further clarified,various therapies target bile acid homeosis and immune dysfunction are being studied.Both to guide the discovery of new therapies and to inform clinicians so that rational treatment regimens can be tailored to patients.