University of Miami Miller School of Medicine, 1500 Northwest, 12th Avenue, Suite 1101, Miami, FL 33136, USA.
Division of Hepatology, University of Miami Miller School of Medicine, University of Miami, 1500 Northwest, 12th Avenue, Suite 1101, Miami, FL 33136, USA.
Clin Liver Dis. 2016 Feb;20(1):113-30. doi: 10.1016/j.cld.2015.08.006.
All patients with primary biliary cirrhosis (PBC) and abnormal liver biochemistry should be considered for specific therapy. Ursodeoxycholic acid (UDCA) is the only FDA-approved drug for treating PBC. Approximately 40% of patients with PBC respond incompletely to treatment with UDCA, thus having increased risk of death or need for liver transplantation. No second-line therapies for patients with inadequate response to UDCA therapy have been approved. This review provides a current perspective on potential new approaches to treatment in PBC, and highlights some of the challenges we face in evaluating and effectively implementing those treatments.
所有原发性胆汁性胆管炎 (PBC) 患者和肝功能异常者都应考虑进行特异性治疗。熊去氧胆酸 (UDCA) 是唯一获得美国食品药品监督管理局 (FDA) 批准用于治疗 PBC 的药物。大约 40% 的 PBC 患者对 UDCA 治疗反应不完全,因此死亡或需要肝移植的风险增加。对于 UDCA 治疗反应不足的患者,尚无批准的二线治疗方法。本文综述了 PBC 治疗的潜在新方法,并强调了在评估和有效实施这些治疗方法方面我们所面临的一些挑战。