摘要
Background&Aims: We used a multistate modeling approach to assess the effect of ursodeoxycholic acid (UDCA) therapy on the natural course of primary biliary cirrhosis (PBC), which remains controversial. Methods: Our population included 2 62 patients with PBC who had received 13-15 mg/kg UDCA daily for a mean of 8 ye ars (range, 1-22 years). Data were analyzed using a multistate Markov model, wi th histologic stage progression,death, and orthotopic liver transplantation (OLT ) as main end points. Survival without OLT was comparedwith that predicted by th e updated Mayo model and with the expected survival in the control population. R esults: Forty-five patients developed cirrhosis, 20 underwent OLT, and 16 died by the censor date. Ten deaths were due to liver disease. The overall survival r ates were 92%at 10 years and 82%at 20 years. Survival rates without OLT were 8 4%and 66%at 10 and 20 years,respectively, which were slightly lower than the s urvival rate of an age-and sex-matched control population (relative risk [RR], 1.4; P =. 1) but better than the spontaneous survival rate as predicted by the u pdated Mayo model (RR,. 5; P <. 01). The survival rate of patients in stage 1 an d 2 was similar to that in the control population (RR,. 8; P =. 5), whereas the probability of death or OLT remained significantly increased in treated patients in late histologic stages (RR, 2.2; P <. 05). Conclusions:Treatment with UDCA a lone normalizes the survival rate of patients with PBC when given at early stage s. However, there is a continued need f- or new therapeutic options in patients with advanced disease.
Background&Aims: We used a multistate modeling approach to assess the effect of ursodeoxycholic acid (UDCA) therapy on the natural course of primary biliary cirrhosis (PBC), which remains controversial. Methods: Our population included 2 62 patients with PBC who had received 13-15 mg/kg UDCA daily for a mean of 8 ye ars (range, 1-22 years). Data were analyzed using a multistate Markov model, wi th histologic stage progression,death, and orthotopic liver transplantation (OLT ) as main end points. Survival without OLT was comparedwith that predicted by th e updated Mayo model and with the expected survival in the control population. R esults: Forty-five patients developed cirrhosis, 20 underwent OLT, and 16 died by the censor date. Ten deaths were due to liver disease. The overall survival r ates were 92%at 10 years and 82%at 20 years. Survival rates without OLT were 8 4%and 66%at 10 and 20 years,respectively, which were slightly lower than the s urvival rate of an age-and sex-matched control population (relative risk [RR], 1.4; P =. 1) but better than the spontaneous survival rate as predicted by the u pdated Mayo model (RR,. 5; P <. 01). The survival rate of patients in stage 1 an d 2 was similar to that in the control population (RR,. 8; P =. 5), whereas the probability of death or OLT remained significantly increased in treated patients in late histologic stages (RR, 2.2; P <. 05). Conclusions:Treatment with UDCA a lone normalizes the survival rate of patients with PBC when given at early stage s. However, there is a continued need f- or new therapeutic options in patients with advanced disease.
