Ursodeoxycholic acid and methotrexate for primary sclerosing cholangitis: A pilot study

Keith D. Lindor, Roberta A. Jorgensen, Monte L. Anderson, Gregory J. Gores, Alan F. Hofmann, Nicholas F. LaRusso

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88 Scopus citations


Objective: Ursodeoxycholic acid (UDCA) and methotrexate (MTX) are both undergoing evaluation for the treatment of patients with primary sclerosing cholangitis (PSC). In this pilot study, we sought to study the safety and estimate of efficacy of a combination of these two drugs administered over a 2-yr period in patients with PSC. Methods: Nineteen patients with well defined PSC were entered prospectively into a pilot study with anticipation of 2-yr follow-up. The patients received UDCA (13-15 mg/kg/day) in divided doses in conjunction with MTX (0.25 mg/kg/wk). The results of treatment were compared with a concurrently studied, but not randomized, group of 10 patients receiving UDCA alone. At entry, the two groups were comparable with respect to age, sex, liver biochemistries, and histological stage when available. Results: During this period, five patients treated with the combination of UDCA and MTX were severed from the study (three referred for transplantation, one death from small bowel cancer, and one with pre- existing, high ileostomy output who withdrew voluntarily). MTX was discontinued by the investigators in an additional five patients (hair loss in three, pulmonary problems in two). There was no change in fatigue or itching compared with baseline in the group receiving the UDCA/MTX combination. Changes in biochemistries from baseline values were not different in the group receiving UDCA and MTX compared with the group receiving UDCA alone. Furthermore, after MTX was withdrawn and UDCA was continued, there was no clear evidence of further biochemical change. The use of MTX in combination with UDCA was associated with toxicity without any further improvement in liver biochemistries compared with the use of UDCA alone. Conclusion: This pilot study found no evidence to support the use of MTX in combination with UDCA for patients with PSC.

Original languageEnglish (US)
Pages (from-to)511-515
Number of pages5
JournalAmerican Journal of Gastroenterology
Issue number3
StatePublished - Mar 1996

ASJC Scopus subject areas

  • Hepatology
  • Gastroenterology


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