Shuhei Hige, Mototsugu Kato, Koji Ogawa, Masato Nakai, Masahiro Asaka, Hiroki Shirato, Hiroshi Taguchi, Hiromasa Horimoto, Takuya Sho, Yusuke Sakuhara, Makoto Chuma, Tomoe Kobayashi, Mitsuru Nakanishi, Daisuke Abo, Katsumi Terashita, Shinichi Shimizu, Yoko Tsukuda, Seiji Tsunematsu, and Yoshiya Yamamoto
Background and Aim: To evaluate the efficacy of intra-arterial 5-fluorouracil (5-FU) and subcutaneous interferon (IFN) combined with image-guided radiation therapy (IGRT) in advanced hepatocellular carcinoma (HCC) with portal vein tumor thrombosis (PVTT). Methods: Twenty HCC patients with PVTT were treated with 5-FU and IFN combined with image-guided radiation therapy (IGRT) (IGRT group), and as controls, 20 patients with PVTT were treated with 5-FU and IFN alone (non-IGRT group). Overall survival (OS) time, response rates, time to progression (TTP) and safety were compared across groups. Results: Complete response (CR), partial response (PR), stable disease (SD) and progressive disease (PD) of PVTT were 5%, 55%, 40% and 0% in the IGRT group and 0%, 30%, 35% and 35%, in the non-IGRT group, respectively. CR, PR, SD, and PD of the whole tumor were 0%, 35%, 45% and 20% in the IGRT group and 0%, 30%, 35% and 35%, in the non-IGRT group, respectively. Overall median survival was significantly longer in the IGRT group (12.0 months 95% confidence interval [CI], 9.3–17.6 months) than in the non-IGRT group (9.1 months [95% CI, 5.5–11.1 months]) (P = 0.041). TTP was significantly longer in the IGRT group (6.9 months [95% CI, 5.6–10.2 months]) than in the non-IGRT group (4.0 months [95% CI, 3.3–6.4 months]) (P = 0.034). Conclusions: The response rates, median OS time and TTP in patients with advanced HCC with PVTT who received this novel combination therapy of intra-arterial 5-FU and subcutaneous IFN with IGRT are encouraging, and this combination therapy warrants further investigation.