Primary Biliary Cholangitis: advances in management and treatment of the disease
Primary Biliary Cholangitis: advances in management and treatment of the disease
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DOI：
10.1016/j.dld.2017.05.001
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发表时间：
2017-08-01
影响因子：
4.5
通讯作者：
Alvaro, Domenico
中科院分区：
文献类型：
--
作者：
Invernizzi, Pietro;Floreani, Annarosa;Alvaro, Domenico
原发性胆汁性胆管炎，以前称为原发性胆汁性肝硬化，是一种罕见疾病，主要影响五至七十岁的女性。它是一种慢性自身免疫性疾病，其特征是小叶间胆管进行性损伤，导致胆管减少、慢性胆汁淤积和胆汁酸潴留。即使这种疾病通常表现出较长的无症状期和缓慢的进展，但在许多患者中，它可能会更快地进展为肝硬化及其并发症。 10年死亡率高于人类免疫缺陷病毒/丙型肝炎病毒混合感染和乳腺癌等疾病。熊去氧胆酸是目前唯一可用的治疗方法，但即使可以有效阻止大多数患者的疾病进展，但约 40% 的患者无法有效降低碱性磷酸酶（疾病活动的替代标志物）。最近，奥贝胆酸获得欧洲药品管理局有条件批准，作为对熊去氧胆酸无反应或不耐受的患者的补充治疗。本文阐述了由具有原发性胆汁性胆管炎专业知识的临床药理学家、胃肠病学家/肝病学家和患者协会组成的工作组对疾病管理的最新技术和未来前景的看法。该文件的一致意见是通过专家会议达成的。 (C) 2017 年作者。由 Elsevier Ltd 代表 Editrice Gastroenterologica Italiana S.r.l 出版。
Primary Biliary Cholangitis, previously known as Primary Biliary Cirrhosis, is a rare disease, which mainly affects women in their fifth to seventh decades of life. It is a chronic autoimmune disease characterized by a progressive damage of interlobular bile ducts leading to ductopenia, chronic cholestasis and bile acids retention. Even if the disease usually presents a long asymptomatic phase and a slow progression, in many patients it may progress faster toward cirrhosis and its complications. The 10 year mortality is greater than in diseases such as human immunodeficiency virus/Hepatitis C Virus coinfection and breast cancer. Ursodeoxycholic acid is the only treatment available today, but even if effective in counteracting the disease progression for the majority of patients, in approximately 40% is not able to decrease effectively the alkaline phosphatase, a surrogate marker of disease activity. Recently, obeticholic acid received the European Medicines Agency conditional approval, as add on treatment in patients non responders or intolerant to ursodeoxycholic acid. The present paper illustrates the opinion of a working group, composed by clinical pharmacologists, gastroenterologists/hepatologists with specific expertise on Primary Biliary Cholangitis and patient associations, on the state of the art and future perspectives of the disease management. The agreement on the document was reached through an Expert Meeting. (C) 2017 The Authors. Published by Elsevier Ltd on behalf of Editrice Gastroenterologica Italiana S.r.l.