Treatment of primary sclerosing cholangitis with low-dose ursodeoxycholic acid: results of a retrospective Italian multicentre survey.

Background. Data concerning the usefulness and type of drugs employed to treat patients with primary sclerosing cholangitis are controversial. Ursodeoxycholic acid has been shown to be a useful agent, however the drug dosage and its effect on the clinical course are still under debate. Aim. To evaluate the efficacy of low-dose ursodeoxycholic acid in the treatment of primary sclerosing cholangitis. Methods. We retrospectively analysed data from 86 patients with primary sclerosing cholangitis from eight centres in Italy between 1987 and 1997: 69 were treated with ursodeoxycholic acid (8–13 mg/kg/day), while 17 received symptomatic treatment and served as controls. The effect of therapy was evaluated by standard liver function tests and symptom analysis. Results. Ursodeoxycholic acid treatment was associated with significant improvement in serum alkaline phosphatase (735±833 vs. 519±448 U/l, p<0.001), γ-glutamyl transpeptidase (401±352 vs. 234±235 U/l, p<0.001), aspartate aminotransferase (87±70 vs. 56±42 U/l, p=0.001), alanine aminotransferase (146±139 vs. 76±73 U/l, p<0.001), and total bilirubin (1.88±2.44 vs. 1.76±4.12 U/l, p=0.01); there was also amelioration of fatigue (p=0.007), jaundice (p=0.002), and body weight loss (p=0.002). Conclusions. Ursodeoxycholic acid, at a dose of 8–13 mg/kg/day was beneficial for the general condition and liver biochemistry of patients with primary sclerosing cholangitis; high-dose ursodeoxycholic acid treatment requires further evaluation.