Background and objective: After tolvaptan was approved for the treatment of cirrhosis patients with ascites, only a few studies have reported its efficacy in the real clinical setting, and no studies have reported its contribution to overall survival. This study clarified the clinical outcomes of tolvaptan treatment in terms of improving ascites unresponsive to standard diuretics (AUS) and overall survival.
Methods: We retrospectively enrolled 80 decompensated cirrhosis patients with AUS who were administered tolvaptan from October 2012 to December 2014. The patients were divided into two groups according to ascites improvement. We compared laboratory results and overall survival and analyzed factors that affected overall survival.
Results: Of the 80 patients, 59 (73.8 %) were male and the median age was 70 years. Thirty-nine (48.8 %) patients were Child-Pugh class C, and 36 (45.0 %) had advanced hepatocellular carcinoma (HCC). Tolvaptan was effective in 48 (60.0 %) patients with an average 3.8 kg weight reduction and ineffective in 32 (40.0 %) patients. The cumulative survival rate differed significantly between the two groups (p < 0.0001), with 87.5 and 68.0 % survival at 30 and 90 days, respectively, in the effective group, and 50.0 and 30.5 % survival, respectively, in the ineffective group. Multivariate analysis showed that improvement in AUS, advanced HCC, total bilirubin level, blood urea nitrogen level, and the presence of hyponatremia were independent predictors of overall survival.
Conclusions: Tolvaptan could possibly improve overall survival in decompensated cirrhosis patients with AUS as long as it was demonstrated to be effective in these patients.