To evaluate the efficacy and safety of arsenic trioxide transarterial chemoembolization and intravenous administration in unresectable hepatocellular carcinoma with lung metastasis.

A single-blind, two-parallel group, randomized trial was conducted at three medical centers (Guangzhou, China), including patients with both biopsy-confirmed hepatocellular carcinoma and lung metastasis. The experimental group received arsenic trioxide transarterial chemoembolization and intravenous administration of arsenic trioxide, while the control group only received arsenic trioxide transarterial chemoembolization. We compared overall survival (OS), time to progression (TTP), disease control rate (DCR), and objective response rate (ORR) between the groups.

Between April 2013 and June 2014, 139 patients received the allocated intervention, 70 of whom were in the experimental group and 69 of whom were in the control group. No patient was lost to follow-up. The median OS was 7.3 (95 % CI = 6.8–7.8) months in the experimental group and 2.9 (95 % CI = 2.6–3.1) months in the control group (P < 0.001). The median TTP was 2.7 (95 % CI = 1.9–3.3) months in the experimental group and 1.2 (95 % CI = 1.0–1.9) months in the control group (P = 0.023). In the experimental group, the DCR was 72.85 % and the ORR was 7.14 %, while in the control group, the DCR was 7.24 % and the ORR was 0.00 %. DCR and ORR differed significantly between the two groups (P < 0.001 and P = 0.024, respectively).

Arsenic trioxide transarterial chemoembolization and intravenous administration were safe and effective in unresectable hepatocellular carcinoma with lung metastasis.