TY - JOUR
T1 - High-dose ursodeoxycholic acid as a therapy for patients with primary sclerosing cholangitis
AU - Harnois, Denise M.
AU - Angulo, Paul
AU - Jorgensen, Roberta A.
AU - Larusso, Nicholas F.
AU - Lindor, Keith D.
PY - 2001/5
Y1 - 2001/5
N2 - 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.
AB - 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.
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U2 - 10.1016/S0002-9270(01)02340-1
DO - 10.1016/S0002-9270(01)02340-1
M3 - Article
C2 - 11374699
AN - SCOPUS:0034998596
SN - 0002-9270
VL - 96
SP - 1558
EP - 1562
JO - American Journal of Gastroenterology
JF - American Journal of Gastroenterology
IS - 5
ER -