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 th e treatment of Chinese patients with primary biliary cirrhosis, a shor t-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 f ollowed by a placebo for 3 months or a placebo for 3 months followed b y UDCA for 3 months. The clinical symptoms of pruritus improved when t he patients were receiving UDCA but became worse when receiving a plac ebo. Mean serum levels of alkaline phosphatase (ALPase), gamma-glutamy l transferase (gamma-GT), total bilirubin, cholesterol, alanine aminot ransferase (ALT) and aspartate aminotransferase all decreased below th e baseline values when receiving UDCA treatment and all increased abov e the baseline values when receiving the placebo. The difference was s tatistically significant. In the second part of the study, 19 patients received long-term UDCA treatment (mean 20 months). The clinical symp toms of pruritus improved in 90% of the pruritic patients. Serum level s 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 UDC A treatment but did not reach statistical significance at the last fol low up. No patient lost antimitochondrial antibody and elevated immuno globulin levels did not improve significantly, but the Mayo clinical r isk score improved significantly after long-term UDCA treatment. Treat ment failure was noted in three patients: two patients in the histolog ic 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 progres sive jaundice. No severe adverse reaction was noted during UDCA treatm ent, only one patient suffered from a mild allergic reaction. In concl usion, UDCA is safe and effective in the treatment of Chinese PBC pati ents in stages I-III.