Background The prognosis of
hepatocellular carcinoma (HCC) with major portal vein
tumor thrombosis (PVTT) is dismal after standard treatment with
sorafenib. Hepatic arterial infusion
chemotherapy (HAIC) has been suggested for patients with HCC and major PVTT. Purpose To compare the efficacy and safety of
sorafenib plus 3cir-OFF HAIC versus
sorafenib alone for advanced HCC with major PVTT. Materials and Methods This phase II trial recruited systemic treatment-naïve patients with HCC and major PVTT (portal vein invasion grade Vp3 [first branch] and Vp4 [main trunk]) between June 2017 and November 2019. Patients were randomly assigned (1:1 ratio) to receive
sorafenib (400 mg twice daily) plus 3cir-OFF HAIC (35 mg/m2
oxaliplatin [hours 0-2] followed by 600 mg/m2
5-fluorouracil [hours 2-24], days 1-3) with a standardized percutaneous
port catheter system or
sorafenib alone (400 mg twice daily) every 4 weeks. The primary end point was overall survival (OS). The secondary end points were objective response rate, progression-free survival (PFS), and safety. OS and PFS were assessed using the Kaplan-Meier method and log-rank test. Results The intent-to-treat population included 64 patients, with 32 in each group. The median OS was 16.3 months (95% CI: 0.0, 35.5) with
sorafenib plus HAIC and 6.5 months (95% CI: 4.4, 8.6) with
sorafenib alone (hazard ratio [HR] = 0.28; 95% CI: 0.15, 0.53; P < .001). A higher objective response rate (41% [n = 13] vs 3% [n = 1], P < .001) and a longer median PFS (9.0 months vs 2.5 months; HR = 0.26; 95% CI: 0.15, 0.47; P < .001) were observed in the
sorafenib plus HAIC group. Grade 3 or 4 adverse events were more frequent in the
sorafenib plus HAIC group, including
diarrhea (n = 7 [22%] vs n = 5 [16%]),
hand-foot syndrome (n = 6 [19%] vs n = 2 [6%]), and
thrombocytopenia (n = 7 [22%] vs n = 0). Conclusion
Sorafenib plus 3cir-OFF hepatic arterial infusion
chemotherapy may be a promising treatment in patients with
hepatocellular carcinoma and major portal vein
tumor thrombosis because of the improved survival and an acceptable safety profile. Clinical trial registration no. NCT03009461 © RSNA, 2022 Online supplemental material is available for this article. See also the editorial by Chung in this issue.