• DOI:10.1053/J.GASTRO.2005.08.017
• Corpus ID: 14982846

High-dose ursodeoxycholic acid in primary sclerosing cholangitis: a 5-year multicenter, randomized, controlled study.

@article{Olsson2005HighdoseUA,
  title={High-dose ursodeoxycholic acid in primary sclerosing cholangitis: a 5-year multicenter, randomized, controlled study.},
  author={Rolf Olsson and Kirsten Muri Boberg and O S de Muckadell and Stefan C. Lindgren and Rolf Hultcrantz and Geir Folvik and Helge Bell and Magnhild Gangs{\o}y-Kristiansen and Jon Matre and Andreas Rydning and Ola Wikman and {\AA}ke Danielsson and Hanna Sandberg-Gertz{\'e}n and K A Ung and Anders S Eriksson and Lars L{\"o}{\"o}f and Hanne Prytz and Hanns-Ulrich Marschall and Ulrika Broomé},
  journal={Gastroenterology},
  year={2005},
  volume={129 5},
  pages={
          1464-72
        }
}

• R. Olsson, K. Boberg, +16 authors U. Broomé
• Published 1 November 2005
• Medicine
• Gastroenterology

BACKGROUND & AIMS
There is no medical treatment of proven benefit for primary sclerosing cholangitis. This study aimed at studying the effect of a higher dose of ursodeoxycholic acid than previously used on survival, symptoms, biochemistry, and quality of life in this disease.

METHODS
A randomized placebo-controlled study was performed in tertiary and secondary gastroenterology units. A total of 219 patients were randomized to 17 to 23 mg/kg body weight per day of ursodeoxycholic acid (n = 110) or placebo (n = 109) for 5 years. Follow-up data are available from 97 patients randomized to ursodeoxycholic acid and for 101 randomized to placebo. Quality of life was assessed by using the Medical Outcomes Study 36-item Short-Form Health Survey.

RESULTS
The combined end point "death or liver transplantation" occurred in 7 of 97 (7.2%) patients in the ursodeoxycholic acid group vs 11 of 101 (10.9%) patients in the placebo group (P = .368; 95% confidence interval, -12.2% to 4.7%). The occurrence of liver transplantation as a single end point showed a similar positive trend for ursodeoxycholic acid treatment (5/97 [5.2%] vs 8/101 [7.9%]; 95% confidence interval, -10.4% to 4.6%). Three ursodeoxycholic acid and 4 placebo patients died from cholangiocarcinoma, and 1 placebo patient died from liver failure. Alkaline phosphatase and alanine aminotransferase tended to decrease during the first 6 months. There were no differences between the 2 groups in symptoms or quality of life. Analyses of serum ursodeoxycholic acid concentration gave no evidence that noncompliance may have influenced the results.

CONCLUSIONS
This study found no statistically significant beneficial effect of a higher dose of ursodeoxycholic acid than previously used on survival or prevention of cholangiocarcinoma in primary sclerosing cholangitis.