Sustained Biochemical and Histologic Remission of Primary Biliary Cirrhosis in Response to Medical Treatment

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Abstract
Treatment of primary biliary cirrhosis with ursodiol or colchicine may stabilize the disease or slow its rate of progression, but no reports of spontaneous or treatment-related remission have been published. To determine whether primary biliary cirrhosis fully responds to low-dose oral methotrexate therapy. Prospective case study with at least 6 years of observation. Academic medical center. 5 of 19 patients with biopsy-proven precirrhotic primary biliary cirrhosis who had been ill for at least 1 year. Three of the 5 had not responded to colchicine or had responded only partially. Oral methotrexate, 15 mg/wk in divided doses. Symptoms, biochemical tests of liver function, and percutaneous liver biopsies. The latter were done at baseline, 1 to 2 years after initiation of methotrexate therapy, and then every 2 to 3 years during methotrexate therapy. All 5 patients completely responded to medical treatment. Results of biochemical tests of liver function became normal, symptoms remitted, and serial liver biopsy specimens showed progressive histologic improvement. Biopsy specimens obtained after 5 to 12 years of treatment showed few signs of primary biliary cirrhosis and, in 3 patients, were close to normal. Five of the other 14 patients have responded biochemically and have shown histologic improvement; the other 9 have not responded to methotrexate therapy, have discontinued therapy, or have been lost to follow-up. In some patients, primary biliary cirrhosis may remit in response to methotrexate alone or in combination with colchicine or ursodiol.

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