原发性胆汁性胆管炎(以前称为原发性胆汁性肝硬化,PBC)是一种以免疫介导的胆管上皮细胞损伤为循环的自身免疫性肝病,胆汁淤积和进行性纤维化可随着时间的推移最终导致终末期胆汁性肝硬化.遗传和环境影响都被认为与疾病的发生有关。PBC在女性和50岁以上的人群中最普遍,但在全球范围内,成年患者中的疾病谱得到认可;男性,发病年龄较小(<45岁)和就诊时疾病进展是预后较差的基线预测因素.随着越来越多地通过胆汁淤积性血清肝脏测试和抗线粒体抗体的存在来诊断该疾病,大多数患者没有肝硬化,术语胆管炎更准确。病程经常伴随着对患者来说可能是负担的症状,PBC患者的管理必须解决,以终身的方式,疾病进展和症状负担。许可治疗包括熊去氧胆酸(UDCA)和奥贝胆酸(OCA),以及实验性的新的和重新利用的药物。疾病管理侧重于对所有患者启动UDCA,并根据基线和治疗因素进行风险分层。特别是对治疗的反应。那些对UDCA治疗不耐受或UDCA治疗失败(通常在试验和临床实践中反映为碱性磷酸酶>1.67×正常和/或胆红素升高的上限)证明为高风险疾病的患者,应考虑用于二线治疗。其中OCA是目前唯一获得许可的国家健康与护理卓越研究所推荐的代理商。患者的随访是终身的,必须解决疾病的治疗和相关症状的管理。
Primary biliary cholangitis (formerly known as primary biliary cirrhosis, PBC) is an autoimmune liver disease in which a cycle of immune mediated biliary epithelial cell injury, cholestasis and progressive fibrosis can culminate over time in an end-stage biliary cirrhosis. Both genetic and environmental influences are presumed relevant to disease initiation. PBC is most prevalent in women and those over the age of 50, but a spectrum of disease is recognised in adult patients globally; male sex, younger age at onset (<45) and advanced disease at presentation are baseline predictors of poorer outcome. As the disease is increasingly diagnosed through the combination of cholestatic serum liver tests and the presence of antimitochondrial antibodies, most presenting patients are not cirrhotic and the term cholangitis is more accurate. Disease course is frequently accompanied by symptoms that can be burdensome for patients, and management of patients with PBC must address, in a life-long manner, both disease progression and symptom burden. Licensed therapies include ursodeoxycholic acid (UDCA) and obeticholic acid (OCA), alongside experimental new and re-purposed agents. Disease management focuses on initiation of UDCA for all patients and risk stratification based on baseline and on-treatment factors, including in particular the response to treatment. Those intolerant of treatment with UDCA or those with high-risk disease as evidenced by UDCA treatment failure (frequently reflected in trial and clinical practice as an alkaline phosphatase >1.67 × upper limit of normal and/or elevated bilirubin) should be considered for second-line therapy, of which OCA is the only currently licensed National Institute for Health and Care Excellence recommended agent. Follow-up of patients is life-long and must address treatment of the disease and management of associated symptoms.