Background: 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.
Objective: To determine whether primary biliary cirrhosis fully responds to low-dose oral methotrexate therapy.
Design: Prospective case study with at least 6 years of observation.
Setting: Academic medical center.
Patients: 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.
Intervention: Oral methotrexate, 15 mg/wk in divided doses.
Measurements: 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.
Results: 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.
Conclusion: In some patients, primary biliary cirrhosis may remit in response to methotrexate alone or in combination with colchicine or ursodiol.