TRIALS OF THERAPIES FOR PRIMARY BILIARY CIRRHOSIS

原发性胆汁性肝硬化（PBC）是一种进行性肝硬化， 被认为是自身免疫性疾病。其病因 未知以进行性肝内胆汁淤积为特征 由于持续的非化脓性破坏性胆管炎， 影响小的肝内胆管。它影响大约1英寸 2,000名50岁以上的妇女，它是3或4个主要的 在美国进行肝移植的原因几次审判 研究免疫抑制剂在PBC中的作用 包括甲氨蝶呤（MTX）。一项随机试验比较了 1991年开始每周口服MTX 7.5 mg和15 mg的效果 并将于一九九八年完成。28例患者被随机分配至 接受两种剂量之一，并根据 有无症状。治疗导致 血清碱性磷酸酶和氨基转移酶水平和福尔斯 IgM水平正常。43%的患者症状改善 46%的患者和肝脏组织学检查。 与肝脏组织学的更大改善相关， 较低剂量在相似百分比的患者中有效。 延长治疗能够维持改善， 具有临床或组织学检查结果的患者百分比 疾病的改善。这些结果表明， 调整剂量的MTX治疗可能有效， 改善PBC患者的症状和肝脏病理。

Primary biliary cirrhosis (PBC) is a progressive liver disease believed to be of autoimmune nature. Its etiology is unknown. It is characterized by progressive intrahepatic cholestasis as a result of ongoing non-suppurative destructive cholangitis that affects small intrahepatic bile ducts. It affects approximate 1 in 2,000 women above the age of 50 and it is one of the 3 or 4 major reasons for liver transplantation in the US. Several trials have been conducted to study the effect of immunosuppressants in PBC including methotrexate (MTX). A randomized trial comparing the effects of 7.5 vs 15 mg per week of oral MTX was started in 1991 and will be completed in 1998. 28 patients were randomized to receive one of the two doses and stratified according to the presence or absence of symptoms. Therapy led to decreases in serum alkaline phosphatase and aminotransferase levels and falls of IgM levels to normal. Symptoms improved in 43 per cent of patients and liver histology in 46 per cent. The higher dose was associated with greater improvements in liver histology but the lower dose was effective in a similar percentage of patients. Prolonged therapy was able to maintain improvements in the percentage of patients who had a clinical or histological improvement in disease. These results indicate that prolonged therapy with adjusted doses of MTX may be effective in ameliorating symptoms and liver pathology in PBC.