Ursodeoxycholic Acid for Treatment of Enlarged Polycystic Liver.

Patients with autosomal dominant polycystic kidney disease and polycystic liver disease (PLD) often have elevated serum levels of alkaline phosphatase (ALP) and gamma-glutamyl transpeptidase (GGT). Ursodeoxycholic acid (UDCA) is used to treat biliary tract diseases, but its effect on PLD remains unclear. UDCA was administered for 1 year at a dose of 300mg daily to seven PLD patients with elevated ALP or GGT levels who were selected for this treatment by experienced clinicians. Laboratory data and liver volumes were compared among three time points: 1 year before UDCA treatment, at the start of UDCA therapy, and 1 year after the start of therapy. Median GGT did not show a significant change between 1 year before UDCA (180IU/L) and the start of UDCA therapy (209IU/L), but it decreased significantly to 98IU/L after 1 year of UDCA therapy (P=0.015 vs. the start of therapy). ALP showed a significant increase from 1 year before UDCA (456IU/L) to the start of UDCA therapy (561IU/L), and then decreased significantly after 1 year of UDCA therapy (364IU/L). Median liver volume did not show any significant changes among these three time points of assessment. UDCA may be effective for reducing biliary enzyme levels and inhibiting the growth of liver cysts in patients with PLD.