Prediction factors of tolvaptan effectiveness in patients with refractory ascites complicated with hepatocellular carcinoma

OBJECTIVE: Tolvaptan (TVP) is an effective treatment for patients with cirrhotic ascites; however, studies have indicated that a sufficient effect is difficult to obtain in patients with hepatocellular carcinoma (HCC). This study evaluates the efficacy of TVP in patients with HCC with refractory ascites. METHODS: We retrospectively enrolled 32 patients with liver cirrhosis and refractory ascites [mean age: 74 years (range, 47–86 years), men: 78.1% (25/32)]. All patients had HCC and were treated with TVP at our hospital. A TVP responder was defined as a patient who experienced decrease in body weight by ≥1.5 kg within 1 week of treatment. Univariate and multivariate analyses were performed to evaluate clinical and laboratory predictive factors of TVP response. RESULTS: The TVP response rate was 46.9% (15/32 patients) after 1 week of treatment. HCC treatment (transcatheter arterial chemoembolization and/or radiofrequency ablation) was administered to 11/15 (73.3%) responders. In the multivariate analysis, the reduction of urine osmolality was higher in responders than nonresponders (202 mOsm/l vs. 65 mOsm/l, P = 0.040), and the tumor stage (P = 0.043) was worse in nonresponders. Multivariate Cox proportional hazards regression analysis indicated a significantly better prognosis among responders than among nonresponders (P < 0.01). CONCLUSION: The HCC tumor stage and the reduction in urine osmolality can predict the efficacy of TVP in patients with refractory ascites complicated with HCC. TVP may allow therapeutic intervention for HCC and improve prognosis, even in patients with Child–Pugh class C.