High-dose ursodeoxycholic acid as a therapy for patients with primary sclerosing cholangitis

Denise M. Harnois, Paul Angulo, Roberta A. Jorgensen, Nicholas F. Larusso, Keith D. Lindor

Research output: Contribution to journalArticle

198 Scopus citations

Abstract

OBJECTIVES: To assess the tolerability and efficacy of high-dose (25-30 mg/kg per day) ursodeoxycholic acid (UDCA) in patients with primary sclerosing cholangitis (PSC). METHODS: Thirty patients with PSC were enrolled in this pilot study and treated for 1 yr. Changes in the Mayo risk score at 1 yr of treatment and projected survival at 4 yr were compared with that observed in patients randomized to placebo (n = 52) or UDCA (n = 53) at a dose of 13-15 mg/kg per day. RESULTS: A marked improvement in serum alkaline phosphatase activity (1265 ± 172 vs 693 ± 110 U/L, p < 0.001), AST (161 ± 037 vs 77 ± 13 U/L, p = 0.001), albumin (4.0 ± 0.1 vs 4.2 ± 0.1 g/dl, p = 0.03), and total bilirubin (1.6 ± 0.3 vs 1.3 ± 0.2 mg/dl, p = 0.1) occurred at 1 yr of therapy with high-dose UDCA. Changes in the Mayo risk score after 1 yr of treatment were significantly different among the three groups (p < 0.001), and these changes would be translated into a significantly different expected survival at 4 yr (p = 0.05). This expected survival at 4 yr was significantly different between placebo and the dose of 25-30 mg/kg per day (p = 0.04), but not between placebo and the dose of 13-15 mg/kg per day (p = 0.4). High-dose UDCA was well tolerated. CONCLUSIONS: UDCA at a dose of 25-30 mg/kg per day may be of benefit for patients with PSC, and this regimen deserves further evaluation in a long-term, randomized, placebo-controlled trial.

Original languageEnglish (US)
Pages (from-to)1558-1562
Number of pages5
JournalAmerican Journal of Gastroenterology
Volume96
Issue number5
DOIs
StatePublished - May 2001
Externally publishedYes

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ASJC Scopus subject areas

  • Hepatology
  • Gastroenterology

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