Survival Benefit of Combined Systemic and Locoregional Therapy in Hepatocellular Carcinoma with Portal Vein Tumor Thrombus: A Propensity Score-Matched Analysis.
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INTRODUCTION: The prognosis of hepatocellular carcinoma (HCC) with portal vein tumor thrombus (PVTT) remains dismal. Although systemic therapy is the standard of care, its effectiveness is limited. This study aimed to compare the efficacy and safety of transarterial chemoembolization (TACE) or hepatic arterial infusion chemotherapy (HAIC) combined with systemic therapy versus systemic therapy alone in patients with HCC and PVTT. METHODS: We retrospectively analyzed 478 patients newly diagnosed with HCC and PVTT between January 2021 and December 2024. Propensity score matching (PSM) was used to balance baseline characteristics. Outcomes compared between the combination therapy group (TACE/HAIC plus targeted therapy and immunotherapy, n = 374) and the systemic therapy group (n = 104) included overall survival (OS), progression-free survival (PFS), tumor response, and adverse events. RESULTS: After PSM (184 vs. 102 patients), the combination therapy group showed significantly longer median OS (15.7 vs. 5.9 months; hazard ratio [HR] = 0.524, 95% confidence interval [CI]: 0.391-0.702; p < 0.001) and PFS (7.0 vs. 3.6 months, HR = 0.732, 95% CI: 0.558-0.959; p = 0.024). The disease control rate was also higher in the combination therapy group (43.5% vs. 27.5%, p = 0.007). Subgroup analyses revealed pronounced survival benefits in patients with Vp4 PVTT and those with Child-Pugh B liver function. Although adverse events were more frequent in the combination therapy group, the incidence of grade 3-4 toxicities was generally comparable between the two groups. CONCLUSION: In HCC patients with PVTT, combining TACE or HAIC with systemic therapy significantly improves survival outcomes compared to systemic therapy alone, with acceptable safety. This multimodal approach offers a promising treatment strategy, particularly for patients with advanced PVTT or impaired liver function.