熊去氧胆酸应答欠佳的原发性胆汁性胆管炎的药物治疗进展

Research Advances in Pharmacotherapy of Primary Biliary Cholangitis with Poor Ursodeoxycholic Acid Response

对一线药物熊去氧胆酸生化应答欠佳的原发性胆汁性胆管炎(PBC)患者的预后较差,这类患者发生肝脏相关疾病和死亡风险大大增加,非移植生存率显著降低。近年来,随着基于生化参数的预后评分模型的开发、应用,不必须通过肝脏活组织检查识别PBC患者已经成为可能。但目前针对此类患者的治疗策略仍缺乏统一共识,包括奥贝胆酸在内的二线疗法及正在开发中的备选药物仍需进一步评估,其疗效及安全性仍需更大规模、更长期的随机对照研究验证。

Patients with primary biliary cholangitis(PBC)who have a poor biochemical response to the first-line drug ursodeoxycholic acid have a poor prognosis;these patients are at greatly increased risk of liver-related disease and death,and non-transplant survival rate is significantly reduced.In recent years,with the development and application of prognostic scoring models based on biochemical parameters,it has become possible to identify patients with PBC without having to go through a liver biopsy.However,there is still a lack of consensus on the treatment strategy for these patients,and second-line therapies including obeticholic acid and alternative drugs under development still need further evaluation,and their efficacy and safety still need to be validated in larger,longer-term randomized controlled studies.