Department of Surgery, Oncology and Gastroenterology.
Studiosa senior, University of Padova, Padova.
Curr Opin Gastroenterol. 2021 Mar 1;37(2):99-104. doi: 10.1097/MOG.0000000000000708.
To discuss the most recent data regarding treatment of patients with primary biliary cholangitis (PBC) with inadequate response to ursodeoxycholic acid (UDCA).
Patients with PBC at high-risk of progressive disease are younger, have advanced fibrosis and showed inadequate response to UDCA after 12 months of treatment. These patients need a second-line treatment in addition to UDCA. The goal of therapy should be the normalization of ALP and bilirubin below 0.6 the upper limit of normal. Obeticholic acid (OCA) has proven to be effective in improving surrogate markers of prognosis in PBC, also in real-life cohort. Pruritus is the most frequent adverse event during treatment with OCA. Bezafibrate is another option in patients with inadequate response to UDCA as it was proven to improve surrogate endpoints, pruritus and even, clinical outcome compared with UDCA monotherapy. Finally, budesonide may be considered in patients with marked portal inflammation. Triple therapy with UDCA, OCA and bezafibrate may be considered in patients showing inadequate response to dual therapy.
Patients with PBC need to be evaluated at baseline, and on-treatment, for the risk of progressive disease and eventually treated with second-line therapies in addition to UDCA.
讨论原发性胆汁性胆管炎（PBC）患者对熊去氧胆酸（UDCA）治疗反应不足的最新数据。
有进展性疾病高风险的 PBC 患者更年轻，纤维化程度更严重，并且在接受 UDCA 治疗 12 个月后反应不足。这些患者除了 UDCA 外还需要二线治疗。治疗目标应该是使 ALP 和胆红素恢复正常，低于正常值上限的 0.6 倍。奥贝胆酸（OCA）已被证明可有效改善 PBC 的预后替代指标，在真实队列中也是如此。治疗期间最常见的不良反应是瘙痒。贝特类药物（如非诺贝特）是对 UDCA 反应不足患者的另一种选择，因为它已被证明可改善替代终点、瘙痒，甚至与 UDCA 单药治疗相比，还可改善临床结局。对于有明显门脉炎症的患者，可以考虑使用布地奈德。对于对双重治疗反应不足的患者，可能需要考虑使用 UDCA、OCA 和非诺贝特三联疗法。
PBC 患者需要在基线和治疗期间评估进展性疾病的风险，并最终除 UDCA 外还需要使用二线治疗。
