Methotrexate therapy for primary biliary cirrhosis

Nancy Bach, Carol Bodian, Henry Bodenheimer, Edward Croen, Paul D. Berk, Swan N. Thung, Keith D. Lindor, Thierry Themeau, Fenton Schaffner

Research output: Contribution to journalArticlepeer-review

35 Scopus citations


OBJECTIVE: Preliminary data suggested possible benefits of methotrexate in primary biliary cirrhosis. We assessed the effectiveness of methotrexate use in primary biliary cirrhosis and its tolerance in patients with this disease. METHODS: A total of 110 primary biliary cirrhosis patients began methotrexate 15 mg/wk; for most, ursodeoxycholic acid was added during the study. We analyzed data from patients completing 5 yr of treatment with methotrexate to assess its effect on biochemical and histologic parameters after 5 yr of therapy. Based on an intent to treat analysis, we also compared survival of our patients (n = 110) with that of patients in a previously published, placebo- controlled trial of ursodeoxycholic acid (n = 180). RESULTS: Only half of the study group completed 5 yr of methotrexate therapy. Therapy did not prevent progression of disease, as indicated by a rising Mayo risk score. Portal fibrosis tended to remain the same. Methotrexate did not diminish the risk of death or liver transplantation when compared with ursodeoxycholic acid or placebo; however, ursodeoxycholic acid use decreased the risk of death or transplant (p = 0.006). CONCLUSIONS: Methotrexate is not well tolerated in primary biliary cirrhosis. The toxicity of methotrexate and its inability to prevent complications of progressive liver disease or improve survival and the need for liver transplantation limits its utility. The benefits of ursodeoxycholic acid were again confirmed.

Original languageEnglish (US)
Pages (from-to)187-193
Number of pages7
JournalAmerican Journal of Gastroenterology
Issue number1
StatePublished - Jan 1 2003
Externally publishedYes

ASJC Scopus subject areas

  • Hepatology
  • Gastroenterology


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