ter Borg Pieter C J, Schalm Solko W, Hansen Bettina E, van Buuren Henk R
Department of Gastroenterology and Hepatology, Erasmus Medical Center, Rotterdam, The Netherlands.
Am J Gastroenterol. 2006 Sep;101(9):2044-50. doi: 10.1111/j.1572-0241.2006.00699.x. Epub 2006 Jul 18.
The therapeutic potential of ursodeoxycholic acid (UDCA) treatment in primary biliary cirrhosis (PBC) remains controversial. In addition, relatively few data have been reported on the outcome of patients who have been treated long term. The aim of the present study was to document long-term survival of a prospectively followed large cohort of UDCA treated patients in comparison to that predicted by the Mayo model and of a matched control cohort of the Dutch population.
Two hundred ninety-seven patients were included and followed during a median period of 68 (range 3-126) months until death or the end of the study.
Survival free of transplantation (1 yr 99.7%, 5 yr 87%, and 10 yr 71%) was significantly better than predicted by the Mayo model (p= 0.01). However, for patients with abnormal serum bilirubin and/or albumin concentrations at entry, observed and predicted survival did not significantly differ. Compared with survival for a standardized cohort of the Dutch population, observed survival for the total group was significantly decreased (p= 0.0003); for noncirrhotic patients and patients with normal entry bilirubin and albumin concentrations survival was comparable. Serum bilirubin and albumin concentrations were the prognostic factors most consistently associated with survival.
A 10-year prognosis for most UDCA-treated patients with PBC, i.e., those with a normal bilirubin and albumin concentration, is comparable to that of a matched general population. Our finding that observed survival was significantly better than predicted by the Mayo model may suggest that this model did not accurately predict prognosis in our cohort. Alternatively, this finding indicates an important therapeutic effect of long-term UDCA treatment in PBC, particularly in patients with noncirrhotic, nonadvanced disease.
熊去氧胆酸（UDCA）治疗原发性胆汁性肝硬化（PBC）的治疗潜力仍存在争议。此外，关于长期接受治疗患者的预后数据报道相对较少。本研究的目的是记录一大群接受UDCA治疗的前瞻性随访患者的长期生存率，并与梅奥模型预测的生存率以及荷兰人群的匹配对照队列进行比较。
纳入297例患者，中位随访时间为68个月（范围3 - 126个月），直至死亡或研究结束。
无移植生存（1年99.7%，5年87%，10年71%）显著优于梅奥模型预测的生存率（p = 0.01）。然而，对于入组时血清胆红素和/或白蛋白浓度异常的患者，观察到的生存率与预测生存率无显著差异。与荷兰人群的标准化队列生存率相比，整个研究组观察到的生存率显著降低（p = 0.0003）；非肝硬化患者以及入组时胆红素和白蛋白浓度正常的患者生存率相当。血清胆红素和白蛋白浓度是与生存最一致相关的预后因素。
大多数接受UDCA治疗的PBC患者，即胆红素和白蛋白浓度正常的患者，其10年预后与匹配的普通人群相当。我们观察到的生存率显著优于梅奥模型预测的生存率这一发现可能表明该模型未能准确预测我们队列中的预后。或者，这一发现表明长期UDCA治疗对PBC具有重要的治疗效果，特别是对非肝硬化、非晚期疾病患者。
