Ursodeoxycholic acid treatment of refractory chronic graft-versus-Host disease of the liver

Ronald H. Fried, Carol S. Murakami, Lloyd D. Fisher, Richard A. Willson, Keith M. Sullivan, George B. McDonald

Research output: Contribution to journalArticlepeer-review

100 Scopus citations


Objective: To determine the safety and efficacy of ursodeoxycholic acid treatment in patients with chronic graft-versus-host disease (GVHD) of the liver. Design: Open-label study in which each patient served as his or her own control. Setting: Private practice and a university bone marrow transplant center. Patients: Twelve patients with refractory chronic GVHD of the liver were studied after allogeneic bone marrow transplantation. Interventions: After baseline data collection, patients were given ursodeoxycholic acid (UDCA, 10 to 15 mg/kg body weight per day) for 6 weeks. After discontinuation of the drug, patients were followed for an additional 6 weeks. Doses of immunosuppressive drugs were unchanged for these 12 weeks. Measurements: Signs, symptoms, Karnofsky performance scores, hematocrit, total leukocyte count, absolute neutrophil count, platelet count, alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase, gamma-glutamyltransferase (GGT), total serum bilirubin, prothrombin time, serum creatinine, and blood urea nitrogen were assessed. Results: Serum tests of cholestatic liver injury measured at 2, 4, and 6 weeks showed improvement compared with baseline. At 6 weeks, the percent decrease from baseline in total serum bilirubin was 33% (P < 0.005); in alkaline phosphatase the decrease was 32% (P < 0.038); and in AST the decrease was 37% (P < 0.007). After discontinuation of UDCA therapy, 11 patients were followed for 6 additional weeks. All showed significant worsening in liver function test results. Symptom scores were unchanged throughout the study. One patient with pruritus improved while receiving therapy with UDCA. No adverse effects were observed. Conclusion: Therapy with UDCA was safe, well-tolerated, and efficacious in the short-term treatment of refractory chronic GVHD of the liver. Further investigation is needed to evaluate the long-term effects of UDCA therapy.

Original languageEnglish (US)
Pages (from-to)624-629
Number of pages6
JournalAnnals of internal medicine
Issue number8
StatePublished - Apr 15 1992

ASJC Scopus subject areas

  • Internal Medicine


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