Nishio A, Keeffe E B, Ishibashi H, Gershwin E M
Department of Gastroenterology, Tenri Hospital, Nara, Japan.
Med Sci Monit. 2000 Jan-Feb;6(1):181-93.
Primary biliary cirrhosis (PBC) is a chronic cholestatic liver disease that predominantly occurs in middle-aged women of various ethnic and racial populations. The disease slowly progresses over decades and is supposed to be caused by immune reactions against host antigens. Histologically, it is characterized by inflammatory destruction of intrahepatic small bile ducts, subsequent fibrosis, and finally liver cirrhosis. It is more frequently diagnosed now than in the past probably because of a greater awareness of the disease. There is only week association of PBC with genetic markers. Liver function tests reveal an elevation of serum alkaline phosphatase and gamma-glutamyl transpeptidase levels with or without elevated aminotransferase levels. The hallmark of the disease is the presence of antimitochondrial antibodies (AMAs), which are found in 95% of patients with PBC. AMAs have been shown to be directed against the 2-oxo-acid dehydrogenase complexes located on the inner membrane of the mitochondria. However, AMA titers do not correlate with the disease severity of progression, and the role of AMAs in the pathogenesis of PBC has not been shown. The disease is frequently associated with other autoimmune diseases, including Sjögren's syndrome, scleroderma and thyroid disorders. Most therapeutic efforts have been directed at altering the immune response. Ursodeoxycholic acid (UDCA) appears to be effective therapy in preventing or delaying the need for liver transplantation and improving survival. However, a number of patients receiving UDCA still develop progressive disease and go on to transplantation, which is an effective therapy at the end stage of the disease. Various prognostic models have been proposed to estimate the survival probability and assist in the determination of the optimum timing of liver transplantation.
原发性胆汁性肝硬化（PBC）是一种慢性胆汁淤积性肝病，主要发生于不同种族和民族的中年女性。该病在数十年间缓慢进展，被认为是由针对宿主抗原的免疫反应所致。组织学上，其特征为肝内小胆管的炎性破坏、随后的纤维化，最终发展为肝硬化。现在比过去更常诊断出该病，这可能是因为对该疾病的认识提高了。PBC与基因标记仅有微弱关联。肝功能检查显示血清碱性磷酸酶和γ-谷氨酰转肽酶水平升高，转氨酶水平可升高或不升高。该病的标志是存在抗线粒体抗体（AMA），95%的PBC患者可检测到该抗体。已证明AMA针对位于线粒体内膜的2-氧代酸脱氢酶复合物。然而，AMA滴度与疾病严重程度或进展并无相关性，AMA在PBC发病机制中的作用尚未明确。该病常与其他自身免疫性疾病相关，包括干燥综合征、硬皮病和甲状腺疾病。大多数治疗措施旨在改变免疫反应。熊去氧胆酸（UDCA）似乎是预防或延迟肝移植需求及提高生存率的有效疗法。然而，一些接受UDCA治疗的患者仍会出现疾病进展并最终接受移植，移植是该病终末期的有效治疗方法。已提出多种预后模型来估计生存概率，并协助确定肝移植的最佳时机。
