Abstract
BACKGROUND: Hepatocellular carcinoma with portal vein tumor thrombus (HCC-PVTT) is a severe condition with poor prognosis. While transarterial chemoembolization (TACE) combined with lenvatinib (TACE-L) shows some promise, survival outcomes remain suboptimal. We hypothesize that TACE-L plus programmed cell death protein-1 inhibitors (TACE-L-P) may offer superior survival benefits compared to TACE-L in this patient population. AIM: To compare efficacy and safety of TACE-L-P vs TACE-L in HCC-PVTT and identify prognostic factors. METHODS: Data from HCC-PVTT patients treated with TACE-L-P or TTACE-L from January 2018 to December 2023 were collected and retrospectively analyzed. Propensity score matching (PSM) method with optimal matching was used to minimize confounding bias. Overall survival (OS), progression-free survival (PFS), objective response rate (ORR), and treatment-related adverse events (AEs) were compared between the two groups. Independent prognostic factors for OS and PFS were elucidated using the Cox proportional hazards model. RESULTS: A total of 100 patients were included, with 42 patients in the TACE-L-P group and 68 patients in the TACE-L group. After PSM performing optimal matching, baseline characteristics were well balanced between the two groups, each comprising 42 patients. The median OS was significantly longer in the TACE-L-P group compared to the TACE-L group (17.2 months vs 12.6 months, P = 0.0207), as was the median PFS (10.6 months vs 7.1 months, P = 0.012). The ORR and disease control rate were both superior in the TACE-L-P group compared to the TACE-L group (66.7% vs 42.9%, P = 0.049; 78.6% vs 50.0%, P = 0.012). Multivariate analysis revealed that the independent prognostic factors for both OS and PFS were the treatment regimen and extrahepatic metastasis. The incidence of any-grade and grade 3 AEs was comparable between the TACE-L-P and TACE-L groups (84.5% vs 88.1%, P = 0.546), with no occurrences of grade 4/5 AEs or treatment-related mortality in either group. CONCLUSION: Compared to TACE-L, the TACE-L-P regimen exhibits an acceptable safety profile and shows potential in improving survival outcomes, making it a promising therapeutic option for patients with HCC-PVTT.