SUSTAINED BIOCHEMICAL AND HISTOLOGIC REMISSION OF PRIMARY BILIARY-CIRRHOSIS IN RESPONSE TO MEDICAL-TREATMENT

Background: Treatment of primary biliary cirrhosis with ursodiol or co lchicine may stabilize the disease or slow its rate of progression, bu t no reports of spontaneous or treatment-related remission have been p ublished. Objective: To determine whether primary biliary cirrhosis fu lly responds to low-dose oral methotrexate therapy. Design: Prospectiv e case study with at least 6 years of observation. Setting: Academic m edical center. Patients: 5 of 19 patients with biopsy-proven precirrho tic primary biliary cirrhosis who had been ill for at least 1 year. Th ree of the 5 had not responded to colchicine or had responded only par tially. Intervention: Oral methotrexate, 15 mg/wk in divided doses. Me asurements: Symptoms, biochemical tests of liver function, and percuta neous liver biopsies. The latter were done at baseline, 1 to 2 years a fter initiation of methotrexate therapy, and then every 2 to 3 years d uring methotrexate therapy. Results: All 5 patients completely respond ed to medical treatment. Results of biochemical tests of liver functio n became normal, symptoms remitted, and serial liver biopsy specimens showed progressive histologic improvement. Biopsy specimens obtained a fter 5 to 12 years of treatment showed few signs of primary biliary ci rrhosis and, in 3 patients, were close to normal. Five of the other 14 patients have responded biochemically and have shown histologic impro vement; the other 9 have not responded to methotrexate therapy, have d iscontinued therapy, or have been lost to follow-up. Conclusion: In so me patients, primary biliary cirrhosis may remit in response to methot rexate alone or in combination with colchicine or ursodiol.