Bhushan Sheena, Sohal Aalam, Kowdley Kris V
Liver Institute Northwest, Seattle, Washington, USA.
Elson S. Floyd College of Medicine, Washington State University, Spokane, Washington, USA .
Am J Gastroenterol. 2025 Jan 1;120(1):151-158. doi: 10.14309/ajg.0000000000003174. Epub 2024 Oct 31.
Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are rare, and chronic cholestatic diseases that can progress to liver failure. The goals of treatment are to halt the progression of liver disease to cirrhosis and/or liver failure, and alleviate symptoms associated with these diseases. Ursodeoxycholic acid has historically been the first-line treatment of PBC, with obeticholic acid and fibrates used as second-line or adjunctive therapies. However, the treatment landscape is rapidly expanding. Recently, 2 new second-line agents gained US Food and Drug Administration approval for the treatment of PBC, and several other therapies remain under investigation with promising results. Although significant progress has been made in the development of therapies for PBC, there are no current approved treatments of PSC other than liver transplantation although several emerging therapies have shown encouraging results. This review outlines the current and upcoming treatments of PBC and PSC.
原发性胆汁性胆管炎(PBC)和原发性硬化性胆管炎(PSC)较为罕见,是可进展为肝衰竭的慢性胆汁淤积性疾病。治疗目标是阻止肝病进展为肝硬化和/或肝衰竭,并缓解与这些疾病相关的症状。熊去氧胆酸历来是PBC的一线治疗药物,奥贝胆酸和贝特类药物用作二线或辅助治疗。然而,治疗方法正在迅速扩展。最近,2种新型二线药物获得了美国食品药品监督管理局批准用于治疗PBC,其他几种疗法仍在研究中,结果令人鼓舞。尽管在PBC治疗方法的研发方面取得了重大进展,但目前除肝移植外,尚无批准用于PSC的治疗方法,不过几种新兴疗法已显示出令人鼓舞的结果。本综述概述了PBC和PSC目前及即将出现的治疗方法。