Background: Hepatocellular carcinoma with inferior vena cava and/or right atrium tumor thrombus (HCC-IVC/RATT) has a poor prognosis and lacks evidence for standard first-line systemic therapy. This study aims to evaluate the effectiveness and safety of three therapeutic regimens in HCC-IVC/RATT: immune checkpoint inhibitors plus molecular-targeted agents (ICI-MTA), hepatic arterial infusion chemotherapy (HAIC), and their combination (ICI-MTA-HAIC).
Methods: This multicenter retrospective cohort study included consecutive HCC-IVC/RATT who received ICI-MTA-HAIC, ICI-MTA, or HAIC from June 2015 to December 2023. Propensity score matching (PSM) was used to balance baseline characteristics.
Results: A total of 355 patients were included: 209 received ICI-MTA-HAIC, 66 received ICI-MTA, and 80 received HAIC. After PSM, the ICI-MTA-HAIC group showed superior median overall survival (OS) to both the ICI-MTA (18.0 vs. 7.5 months, p < 0.001) and HAIC (18.5 vs. 7.1 months, p < 0.001) groups. The ICI-MTA-HAIC group demonstrated better median progression-free survival (PFS) and objective response rate (ORR) compared to the ICI-MTA (PFS: 9.5 vs. 4.4 months; ORR: 47.0% vs. 21.3%, all p < 0.001) and HAIC (PFS: 9.5 vs. 4.4 months; ORR: 48.8% vs. 21.6%, all p < 0.001) groups. There was no significant difference in OS, PFS, or ORR between the ICI-MTA and HAIC groups (all p > 0.05). Grade 3-4 adverse event rates were 49.8%, 33.3%, and 35.0% for the ICI-MTA-HAIC, ICI-MTA, and HAIC groups, respectively. No unexpected events or treatment-related deaths were observed.
Conclusion: ICI-MTA-HAIC was a safe and effective therapy that prolonged the survival of HCC-IVC/RATT compared to ICI-MTA or HAIC.
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