Sustained biochemical and histologic remission of primary biliary cirrhosis in response to medical treatment.

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.