Clinical significance of serum bilirubin levels under ursodeoxycholic acidtherapy in patients with primary biliary cirrhosis

We determined whether the normalization of serum bilirubin level (SBL) indu ced by ursodeoxycholic acid (UDCA) therapy was associated with an improved clinical outcome in patients with primary biliary cirrhosis (PBC), We estim ated the prognostic values of SBL measured after 6 months of UDCA treatment for survival free of orthotopic liver transplantation (OLT). We used a dat abase of 548 patients with PBC followed in three trials of UDCA, Among UDCA -treated patients, we compared survival free of OLT in patients with normal ized SBL (less than or equal to 17 mu mol/L) with those who had persistentl y elevated SBL. Difference in survival was tested between UDCA-treated pati ents whose SBL normalized with treatment and placebo patients who had norma l baseline SBL, We evaluated, in each treatment group, the prognostic value of g-month SBL. Survival was estimated using the Kaplan-Meier method and c ompared by the Cox model. Survival free of OLT was significantly longer in patients who had normalized SBL (P <.0001; relative risk [RR]: 3.7, UDCA gr oup). Survival free of OLT was not significantly different between UDCA pat ients with normalized SBL and placebo patients with a normal baseline SBL ( P =.69). For several cutoffs of 6-month SBL, RRs of OLT or death were simil ar in UDCA-treated and placebo patients: the RR of OLT or death associated with a 6-month SBL more than 30 mu mol/L was 6.0 for UDCA and 5.7 for place bo groups. In conclusion, normalization of SBL during therapy is associated with improved clinical outcome. SBL under UDCA therapy is a prognostic fac tor in PBC. SBL under UDCA therapy should be interpreted as in untreated pa tients.