URSODEOXYCHOLIC ACID IN THE TREATMENT OF PRIMARY BILIARY-CIRRHOSIS - A SHORT-TERM, RANDOMIZED, DOUBLE-BLIND CONTROLLED, CROSS-OVER STUDY WITH LONG-TERM FOLLOW-UP

In order to evaluate the efficacy of ursodeoxycholic acid (UDCA) in the treatment of Chinese patients with primary biliary cirrhosis, a short-term, randomized, double-blind controlled, cross-over study was done with long-term follow up. In the first part of the study, 12 patients were randomly chosen to receive either UDCA 600 mg/day for 3 months followed by a placebo for 3 months or a placebo for 3 months followed by UDCA for 3 months. The clinical symptoms of pruritus improved when the patients were receiving UDCA but became worse when receiving a placebo. Mean serum levels of alkaline phosphatase (ALPase), gamma-glutamyl transferase (gamma-GT), total bilirubin, cholesterol, alanine aminotransferase (ALT) and aspartate aminotransferase all decreased below the baseline values when receiving UDCA treatment and all increased above the baseline values when receiving the placebo. The difference was statistically significant. In the second part of the study, 19 patients received long-term UDCA treatment (mean 20 months). The clinical symptoms of pruritus improved in 90% of the pruritic patients. Serum levels of ALPase, gamma-GT and ALT fell significantly from the pretreatment values 6, 12 and from the mean 20 months after UDCA treatment. Serum levels of total bilirubin fell significantly 6 and 12 months after UDCA treatment but did not reach statistical significance at the last follow up. No patient lost antimitochondrial antibody and elevated immunoglobulin levels did not improve significantly, but the Mayo clinical risk score improved significantly after long-term UDCA treatment. Treatment failure was noted in three patients: two patients in the histologic stage IV with clinical overt jaundice died of complications 4 and 5 months after UDCA treatment, respectively; another patient underwent a liver transplantation 1 year after the UDCA treatment due to progressive jaundice. No severe adverse reaction was noted during UDCA treatment, only one patient suffered from a mild allergic reaction. In conclusion, UDCA is safe and effective in the treatment of Chinese PBC patients in stages I-III.