Angulo P, Lindor K D, Therneau T M, Jorgensen R A, Malinchoc M, Kamath P S, Dickson E R
Division of Gastroenterology and Hepatology, Mayo Clinic and Foundation, Rochester, MN 55905, USA.
Liver. 1999 Apr;19(2):115-21. doi: 10.1111/j.1478-3231.1999.tb00020.x.
BACKGROUND/AIMS: Ursodeoxycholic acid (UDCA) is an effective therapy for most patients with primary biliary cirrhosis (PBC). During the management of these treated patients, a number of clinically important issues arose including which patients might be candidates for combined therapy, which patients require endoscopy for variceal bleeding, and how survival can be predicted during treatment. Our aims were: 1) to identify factors associated with a suboptimal response to UDCA in patients with PBC; 2) to define a simple, non-invasive method to predict those PBC patients most apt to have esophageal varices; and 3) to determine the reliability of the Mayo survival model in predicting the course of UDCA treated patients.
We analyzed the prospectively collected data of 180 patients, who we continue to follow, with PBC who participated in a randomized, placebo-controlled trial of UDCA.
After six months of UDCA therapy, patients with serum alkaline phosphatase levels less than twice normal (p < 0.04), and/or a Mayo risk score < 4.5 (p < 0.04) were more likely to respond favorably to treatment over a two year period. The Mayo risk score was the single risk factor independently predictive of development of varices (p < 0.01); 93% of patients who developed varices had a Mayo risk score > or = 4. The Mayo survival model, recalculated after 6 months on UDCA therapy accurately predicted patient survival.
Suboptimal responders to UDCA can be identified by assessment of serum alkaline phosphatase levels, and/or Mayo risk score. A Mayo risk score above 4 helps in selecting patients for endoscopic surveillance for varices and the Mayo survival model accurately predicts the clinical course in patients with PBC receiving UDCA.
背景/目的：熊去氧胆酸（UDCA）对大多数原发性胆汁性肝硬化（PBC）患者是一种有效的治疗方法。在对这些接受治疗的患者进行管理的过程中，出现了一些临床上重要的问题，包括哪些患者可能适合联合治疗，哪些患者需要进行内镜检查以预防静脉曲张出血，以及如何在治疗期间预测生存率。我们的目的是：1）确定PBC患者中对UDCA反应欠佳的相关因素；2）定义一种简单、非侵入性的方法来预测哪些PBC患者最容易发生食管静脉曲张；3）确定梅奥生存模型在预测接受UDCA治疗患者病程方面的可靠性。
我们分析了180例PBC患者的前瞻性收集数据，这些患者参与了一项UDCA的随机、安慰剂对照试验，我们仍在对其进行随访。
在UDCA治疗6个月后，血清碱性磷酸酶水平低于正常上限两倍（p < 0.04）和/或梅奥风险评分< 4.5（p < 0.04）的患者在两年期间对治疗更可能有良好反应。梅奥风险评分是静脉曲张发生的唯一独立预测风险因素（p < 0.01）；发生静脉曲张的患者中93%的梅奥风险评分≥4。在UDCA治疗6个月后重新计算的梅奥生存模型准确地预测了患者的生存率。
可通过评估血清碱性磷酸酶水平和/或梅奥风险评分来识别对UDCA反应欠佳的患者。梅奥风险评分高于4有助于选择患者进行静脉曲张的内镜监测，并且梅奥生存模型能准确预测接受UDCA治疗的PBC患者的临床病程。
