原发性胆汁性胆管炎药物治疗的基本观念及进展

Pharmacotherapy for primary biliary cholangitis: basic concepts and research advances

原发性胆汁性胆管炎(PBC)是一种原因不明的慢性进行性胆汁淤积性肝病,其特征性表现为高特异性的血清抗线粒体抗体和免疫介导的肝内小胆管非化脓性破坏性炎症,从而导致汇管区炎症与纤维化,最终发展为肝硬化及肝衰竭。熊去氧胆酸(UDCA)是目前唯一公认的用于治疗PBC的药物,推荐剂量为13~15 mg·kg-1·d-1,UDCA治疗获得生化应答患者的生存率可明显改善。然而,大约40%的PBC患者对UDCA治疗无应答,这部分存在疾病进展风险的患者亟需其他药物治疗。基于近年临床研究资料与指南,总结了目前PBC药物治疗的基本观念及最新进展,并展望正在临床试验的治疗新药,以期为UDCA治疗应答欠佳的PBC患者带来新的希望。

Primary biliary cholangitis（ PBC） is a chronic progressive cholestatic liver disease of unknown etiology characterized by highly specific anti-mitochondrial antibody in serum and immune-mediated non-pyogenic destructive infection in the small intrahepatic bile ducts,which can lead to portal inflammation and fibrosis and finally progress to liver cirrhosis and liver failure. At present,ursodeoxycholic acid（ UDCA） is the only drug approved for the treatment of PBC with a recommended dose of 13-15 mg·kg-1·d-1. There are significant improvements in the survival rate of patients achieving biochemical response after UDCA treatment. However,about 40% of PBC patients do not respond to UDCA,and such patients have a risk of disease progression and are in urgent need of other drugs. With reference to recent clinical studies and guidelines,this article summarizes the basic concepts and latest advances in pharmacotherapy for PBC,as well as the perspectives of new drugs in clinical trials,in order to bring new hopes to PBC patients with poor response to UDCA.