The effect of ursodeoxycholic acid therapy on the natural course of primary biliary cirrhosis
The effect of ursodeoxycholic acid therapy on the natural course of primary biliary cirrhosis
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DOI：
10.1053/j.gastro.2004.11.009
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发表时间：
2005-02-01
期刊：
影响因子：
29.4
通讯作者：
Poupon, R
中科院分区：
文献类型：
--
作者：
Corpechot, C;Carrat, F;Poupon, R
背景和目标：我们采用多状态建模方法来评估熊去氧胆酸（UDCA）治疗对原发性胆汁性肝硬化（PBC）自然病程的影响，这一点仍存在争议。方法：我们的人群包括262例PBC患者，他们接受了13-15 mg/kg UDCA每日一次，平均8年（范围：1-22年）。采用多状态马尔可夫模型分析数据，以组织学分期进展、死亡和原位肝移植（奥尔特）作为主要终点。将无奥尔特的生存率与更新的马约模型预测的生存率以及对照人群的预期生存率进行比较。结果：45例患者发展为肝硬化，20例接受奥尔特，16例死亡。10人死于肝病。10年和20年的总生存率分别为92%和82%。10年和20年的非奥尔特生存率分别为84%和66%，略低于年龄和性别匹配的对照人群的生存率（相对危险度[RR]，1.4; P = .1），但优于更新的马约模型预测的自发生存率（RR，.5; P < .01）。1期和2期患者的生存率与对照人群相似（RR，.8; P = .5），而在晚期组织学阶段接受治疗的患者中，死亡或奥尔特的概率仍显著增加（RR，2.2; P < .05）。结论：在早期给予UDCA单独治疗可使PBC患者的生存率正常化。然而，在晚期疾病患者中持续需要新的治疗选择。
Background & Aims: We used a multistate modeling approach to assess the effect of ursodeoxycholic acid (UDCA) therapy on the natural course of primary billary cirrhosis (PBC), which remains controversial. Methods: Our population included 262 patients with PBC who had received 13-15 mg/kg UDCA daily for a mean of 8 years (range, 1-22 years). Data were analyzed using a multistate Markov model, with histologic stage progression, death, and orthotopic liver transplantation (OLT) as main end points. Survival without OLT was compared with that predicted by the updated Mayo model and with the expected survival in the control population. Results: 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 rates were 92% at 10 years and 82% at 20 years. Survival rates without OLT were 84% and 66% at 10 and 20 years, respectively, which were slightly lower than the survival 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 updated Mayo model (RR, .5; P < .01). The survival rate of patients in stage 1 and 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 alone normalizes the survival rate of patients with PBC when given at early stages. However, there is a continued need for new therapeutic options in patients with advanced disease.