Early Biochemical Response to Ursodeoxycholic Acid and Long-Term Prognosis of Primary Biliary Cirrhosis: Results of a 14-Year Cohort Study
Early Biochemical Response to Ursodeoxycholic Acid and Long-Term Prognosis of Primary Biliary Cirrhosis: Results of a 14-Year Cohort Study
复制标题
DOI：
10.1002/hep.26322
复制
发表时间：
2013-07-01
期刊：
影响因子：
13.5
通讯作者：
Zhang, Feng-Chun
中科院分区：
文献类型：
--
作者：
Zhang, Li-Na;Shi, Tian-Yan;Zhang, Feng-Chun
原发性胆汁性肝硬化患者对熊去氧胆酸（UDCA）的生化反应是长期预后的有力预测指标，因此有助于快速识别需要新治疗方法的患者。基于1年UDCA的生化反应，已经研究了许多预测治疗结果的标准。我们试图确定3个月或6个月的早期生化反应是否可以有效地识别预后不良风险的患者，如肝脏相关死亡、肝移植和肝硬化并发症。我们分析了187例患者的前瞻性数据，中位随访时间为5.8年（1.3-14年）。无不良反应的5年和10年生存率分别为86%和63%。在UDCA治疗下，实验室肝脏参数在前3个月得到最显著的改善（P < 0.0001），随后几个月保持相对稳定。巴黎、巴塞罗那、多伦多和爱希姆的定义，而不是鹿特丹的定义，在3、6和12个月时对患者的长期预后有显著的区别。与UDCA治疗1年后的生化反应相比，3个月生化反应的阳性预测值（PPV）较高，阴性预测值（NPV）较低，阴性似然比（NLR）升高；第6个月的生化反应显示PPV和NPV高于或相同，NLR低于所有定义。结论：对于先前公布的标准，第6个月的生化反应可以代替UDCA治疗1年后的评价。我们的发现证明了更快速地识别需要新治疗方法的患者是正确的。
The biochemical response to ursodeoxycholic acid (UDCA) in primary biliary cirrhosis is a strong predictor of long-term outcome and thus facilitates the rapid identification of patients needing new therapeutic approaches. Numerous criteria for predicting outcome of treatment have been studied based on biochemical response to UDCA at 1 year. We sought to determine whether an earlier biochemical response at 3 or 6 months could as efficiently identify patients at risk of poor outcome, as defined by liver-related death, liver transplantation, and complications of cirrhosis. We analyzed the prospectively collected data of 187 patients with a median follow-up of 5.8 years (range, 1.3-14 years). The survival rates without adverse outcome at 5 years and 10 years were 86% and 63%. Under UDCA therapy, laboratory liver parameters experienced the most prominent improvement in the first 3 months (P < 0.0001) and then stayed relatively stable for the following months. The Paris, Barcelona, Toronto, and Ehime definitions, but not the Rotterdam definition, applied at 3, 6, and 12 months significantly discriminated the patients in terms of long-term outcome. Compared with biochemical responses evaluated after 1 year of UDCA therapy, biochemical responses at the third month demonstrated higher positive predictive value (PPV) but lower negative predictive value (NPV) and increased negative likelihood ratio (NLR) by all definitions; biochemical responses at the sixth month showed higher or the same PPV and NPV and lower NLR by all definitions. Conclusion: For the previously published criteria, biochemical responses at the sixth month can be used in place of those evaluated after 1 year of UDCA therapy. Our findings justify a more rapid identification of patients who need new therapeutic approaches.