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

Ann Intern Med. 1997 May 1;126(9):682-8. doi: 10.7326/0003-4819-126-9-199705010-00002.


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.

Publication types

  • Case Reports
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adult
  • Alanine Transaminase / blood
  • Alkaline Phosphatase / blood
  • Bilirubin / blood
  • Cholagogues and Choleretics / therapeutic use*
  • Colchicine / therapeutic use
  • Female
  • Follow-Up Studies
  • Humans
  • Liver Cirrhosis, Biliary / blood
  • Liver Cirrhosis, Biliary / drug therapy*
  • Liver Cirrhosis, Biliary / pathology
  • Male
  • Methotrexate / therapeutic use*
  • Middle Aged
  • Prospective Studies
  • Remission Induction
  • Ursodeoxycholic Acid / therapeutic use


  • Cholagogues and Choleretics
  • Ursodeoxycholic Acid
  • Alanine Transaminase
  • Alkaline Phosphatase
  • Bilirubin
  • Colchicine
  • Methotrexate