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.
Methods: 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.
Results: 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.
Conclusions: 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.