LAUSR

BACKGROUND The long-term survival of patients with hepatocellular carcinoma (HCC) with portal vein tumor thrombus (PVTT) is poor. Systemic therapy, transcatheter arterial chemoembolization (TACE), and hepatic artery infusion chemotherapy (HAIC) are widely used in HCC patients with PVTT. This study aims to explore the efficacy of combining systemic therapy with transarterial-based therapy in HCC patients with PVTT. MATERIALS AND METHODS We retrospectively reviewed data of HCC patients with PVTT treated with combination therapy (TACE-HAIC with tyrosine kinase inhibitors and PD-1 inhibitors) or TACE alone in SYSUCC from 2011 to 2020. The overall survival (OS), progression-free survival (PFS), and overall response rate (ORR) were compared. Propensity score matching (PSM) was used to minimize confounding bias. RESULTS A total of 743 HCC patients with PVTT received combination therapy (n=139) or TACE alone (n=604). After PSM, the ORR was significantly higher in the combination group than in the TACE group (42.1% vs. 5.0%, P < 0.001 [RECIST]; 53.7% vs. 7.8%, P < 0.001 [mRECIST]). The combination group showed significantly better OS than the TACE group (median OS not reached vs. 10.4 months, P < 0.001). The median PFS of the combination and TACE groups was 14.8 and 2.3 months (P < 0.001), respectively. Tumor downstaging followed by salvage liver resection was significantly more common for the combination therapy group than for TACE group (46.3% vs. 4.5%, P < 0.001). After salvage liver resection, 31.6% (30/95) and 1.7% (3/179) of the patients achieved a pathological complete response in the combination and TACE groups, respectively (P < 0.001). The grade 3/4 adverse events rates were similar between the two groups (28.1% vs. 35.9%, P=0.092). CONCLUSION Compared with TACE alone, combination therapy was safer and resulted in survival benefits. This is a promising treatment option for HCC patients with PVTT.