The safety and efficacy of treatment with ursodeoxycholic acid (UDCA) of the patients with primary biliary cirrhosis (PBC) were investigated by following 1462 and 1327 patients, respectively, for 3 to 5 years after the start of treatment. No serious adverse events related to UDCA treatment occurred in the patients investigated and the start of treatment induced the significant fall of serum alkaline phosphatase (Al-P), γGTP, AST, ALT and total bilirubin concentration (T-Bil), which was sustained throughout the investigation period, indicating that the prolonged UDCA treatment was safe and effective in the improvement of liver function tests. In the final visit, however, little or no improvement of Al-P, γGTP, AST, ALT and T-Bil was noticed in some of the patients treated. The stratification of the patients according to the daily dose of UDCA revealed that, in each of the liver function tests, the frequency of those who showed little or no improvement decreased with the increase in the dose, suggesting that the increase in the dose improved the liver function tests in these patients. Twenty three patients died of liver-related causes or received liver transplantation. Multivariate Cox regression analysis indicated that the base line T-Bil greater than 1.2 mg/d
l, advanced histological stage and decrease less than 30% of base line level in T-Bil or AST at 6 months after the start of treatment were the predictors for liver-related death or liver transplantation. The response of T-Bil or AST to UDCA treatment is useful to identify the patients who need additional treatment for improvement of prognosis.
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