Randomized controlled study to examine the efficacy of hepatic arterial infusion chemotherapy with cisplatin before radiofrequency ablation for hepatocellular carcinoma

Aim Hepatic arterial infusion chemotherapy (HAIC) with cisplatin is beneficial to patients with advanced hepatocellular carcinoma (HCC) treated with sorafenib. This study aimed to examine the effect of HAIC with cisplatin before radiofrequency ablation (RFA) in patients with HCC. Methods This was a multicenter, single‐blinded, randomized controlled study (UMIN000007267). Early‐stage HCC patients were randomly assigned (1:1) to receive HAIC with cisplatin before RFA therapy (HAIC group) or RFA monotherapy (non‐HAIC group). The primary end‐point was recurrence‐free survival. Efficacy analysis and safety analysis followed the intention‐to‐treat principle. Results Between August 2012 and July 2016, 74 patients were recruited. A total of 70 eligible patients were randomly assigned to the HAIC group (n = 35) and non‐HAIC group (n = 35). Recurrence‐free survival rates at 1 (3) year in the HAIC group and non‐HAIC group were 82.9% (54.3%) and 74.3% (34.3%), respectively (hazard ratio [HR], 0.597; 95% confidence interval [CI], 0.320–1.091; p = 0.094]. Subgroup analysis showed that the beneficial effect of HAIC was observed in patients with a single nodule and Child–Pugh score 5. Intrahepatic distant recurrence‐free survival rate in the HAIC group was significantly better than that in the non‐HAIC group (HR, 0.468; 95% CI, 0.235–0.896; p = 0.022). Adverse events were observed in just two patients in the HAIC group (6%) – grade 2 cholecystitis and grade 2 hyperkalemia. Conclusions HAIC with cisplatin before RFA did not significantly decrease recurrence in patients with early‐stage HCC. However, it might be effective in preventing intrahepatic distant recurrence.