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

1 May 1997 | Volume 126 Issue 9 | Pages 682-688

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.

Author and Article Information

From New England Medical Center, Tufts University School of Medicine, and the Tupper Research Institute, Boston, Massachusetts.
Acknowledgment: The authors thank Ms. Jane Bankoff Popkin for assistance with manuscript preparation.
Grant Support: In part by General Clinical Research Center Grant RR 00054 from the National Institutes of Health National Center for Research Resources and by GRASP Center grant P30 DK34928 from the National Institute of Diabetes and Digestive and Kidney Diseases.
Requests for Reprints: Marshall M. Kaplan, MD, Gastroenterology Division, Box 233, New England Medical Center, 750 Washington Street, Boston, MA 02111.
Current Author Addresses: Drs. Kaplan, DeLellis, and Wolfe: Gastroenterology Division, Box 233, New England Medical Center, 750 Washington Street, Boston, MA 02111.