Ursodeoxycholic acid: a second look. Primary biliary cirrhosis: dashed hopes

(1) In 1996 the evidence, though limited, suggested that the hydrophilic bile acid ursodeoxycholic acid slowed the progression of primary biliary cirrhosis. Given the absence of an effective alternative, we considered that the treatment was "A real advance" in this setting. (2) The file is far bulkier now. A meta-analysis of 11 randomised placebo-controlled trials of ursodeoxycholic acid in the treatment of primary biliary cirrhosis did not show efficacy. Histologically, the data suggest that the drug lowers the risk of hepatic fibrosis, but this is based on a low level of evidence. Moreover, there was no clinical improvement in the longest randomised trial (median follow-up 3.4 years). (3) Likewise, ursodeoxycholic acid seems to have a biochemical effect but no clear clinical effect in sclerosing cholangitis or in treating the hepatic complications of cystic fibrosis. (4) The main adverse effects are gastrointestinal disorders. (5) There are still no drugs clearly capable of slowing the clinical progression of primary biliary cirrhosis. Nevertheless, given the minor adverse effects of ursodeoxycholic acid, some patients may wish to gamble on a possible benefit in the very long term. (6) When primary biliary cirrhosis becomes symptomatic (with encephalopathy, osteomalacia, etc.) pending liver transplantation, treatment is aimed at controlling symptoms and preventing the rupture of oesophageal varices.