Department of Internal Medicine and Liver Research Institute, Seoul National University College of Medicine, Seoul, Korea.
Department of Internal Medicine, Yonsei University College of Medicine, Yonsei Liver Center, Severance Hospital, Seoul, Korea.
Liver Int. 2019 Sep;39(9):1776-1785. doi: 10.1111/liv.14165. Epub 2019 Jul 2.
BACKGROUND & AIMS: There is no proven treatment for ursodeoxycholic acid (UDCA)-refractory primary biliary cholangitis (PBC) other than obeticholic acid. Although fibrates have been reported to improve biochemical parameters, the long-term effects remain unclear. This study evaluated the effect of fibrate on clinical outcomes of UDCA-refractory PBC.
Patients whose alkaline phosphatase (ALP) was not normalized with at least 13 mg/kg of UDCA treatment for >1 year were included from two tertiary referral centres. The primary outcome was ALP normalization. Secondary outcomes included the development of cirrhosis and hepatic deterioration. Immortal time bias was adjusted using the Mantel-Byar method.
A total of 100 UDCA-refractory PBC patients were included: 71 patients received UDCA alone (the UDCA group) and 29 patients received UDCA plus additional fibrate treatment of 160 mg/d fenofibrate or 400 mg/d bezafibrate (the fibrate/UDCA group). During the follow-up period, the probability of ALP normalization was significantly higher in the fibrate/UDCA group (hazard ratio [HR] = 5.00, 95% confidence interval = 2.87-8.27, P < 0.001). Among 58 non-cirrhotic patients (43 in the UDCA group and 15 in the fibrate/UDCA group), 19 patients (44.1%) in the UDCA group and none in the fibrate/UDCA group developed cirrhosis (HR = 0.12, P = 0.04). Hepatic deterioration (Child-Pugh score increase or signs of decompensated cirrhosis) occurred in 17 patients (23.9%) of the UDCA group and none in the fibrate/UDCA group in which the difference was significant (HR = 0.12, P = 0.04).
In patients with UDCA-refractory PBC, additional fibrate treatment is associated with a higher probability of ALP normalization and a lower risk of cirrhosis development and hepatic deterioration.
熊去氧胆酸(UDCA)难治性原发性胆汁性胆管炎(PBC)除奥贝胆酸外尚无其他有效治疗方法。虽然已有报道称贝特类药物可改善生化参数,但长期疗效仍不清楚。本研究旨在评估贝特类药物对 UDCA 难治性 PBC 临床结局的影响。
从两个三级转诊中心纳入碱性磷酸酶(ALP)在至少 13mg/kg UDCA 治疗 1 年以上仍未正常化的 PBC 患者。主要结局是 ALP 正常化。次要结局包括肝硬化和肝脏恶化的发生。采用 Mantel-Byar 法调整无事件时间偏倚。
共纳入 100 例 UDCA 难治性 PBC 患者:71 例接受 UDCA 单药治疗(UDCA 组),29 例接受 UDCA 加用 160mg/d 非诺贝特或 400mg/d 苯扎贝特(贝特/UDCA 组)。在随访期间,贝特/UDCA 组 ALP 正常化的概率明显更高(风险比[HR] 5.00,95%置信区间 2.87-8.27,P<0.001)。在 58 例非肝硬化患者(UDCA 组 43 例,贝特/UDCA 组 15 例)中,UDCA 组有 19 例(44.1%)患者发生肝硬化,而贝特/UDCA 组无患者发生肝硬化(HR 0.12,P=0.04)。UDCA 组 17 例(23.9%)患者出现肝功能恶化(Child-Pugh 评分增加或失代偿性肝硬化迹象),而贝特/UDCA 组无患者出现肝功能恶化(HR 0.12,P=0.04)。
在 UDCA 难治性 PBC 患者中,加用贝特类药物治疗可提高 ALP 正常化的概率,并降低肝硬化发生和肝功能恶化的风险。