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The Efficacy and Safety of Combined Locoregional and Systemic Therapy versus Systemic Therapy Alone in Patients with Hepatocellular Carcinoma and Portal Vein Tumor Thrombosis: A Multicenter Real-World Retrospective Cohort Study

Year: 2026

Type: article

Abstract: Background: To compare the efficacy and safety of combined locoregional and systemic therapy vs systemic therapy alone in patients with hepatocellular carcinoma (HCC) and portal vein tumor thrombosis (PVTT). Methods: This multicenter retrospective study included 337 patients with HCC and PVTT who received systemic therapy alone (n=121) or combined locoregional and systemic therapy (n=216). Propensity score matching (PSM) was performed to reduce baseline imbalances. Overall survival (OS), progression-free survival (PFS), tumor response and adverse events (AEs) were compared. When the proportional hazards assumption was violated, time-varying Cox and restricted mean survival time (RMST) analyses were performed. Results: In the whole cohort, combined therapy was associated with a lower hazard of death (hazard ratio [HR]=0.57, 95% confidence interval [CI]=0.38– 0.86, P =0.007), and this association remained after PSM (HR=0.54, 95% CI=0.34– 0.86, P =0.009). Conventional Kaplan–Meier analysis did not show a significant PFS difference after PSM (median PFS, 12.97 vs 10.20 months; P =0.394); however, the proportional hazards assumption for PFS was violated. RMST analyses showed significant cumulative PFS advantages favoring combined therapy at 12 months (+1.48 months; P =0.008) and 16 months (+1.69 months; P =0.010). After PSM, the objective response rate according to the modified Response Evaluation Criteria in Solid Tumors (mRECIST) was higher with combined therapy (61.1% vs 45.4%, P =0.029). In patients with Vp3– 4 PVTT, combined therapy was associated with a lower hazard of death (HR=0.60, 95% CI=0.39– 0.93, P =0.023). Grade 3– 4 AEs were comparable between groups (29.2% vs 31.4%, P =0.759). Conclusion: Combined locoregional and systemic therapy was associated with longer OS and an early, time-dependent PFS advantage compared with systemic therapy alone in patients with HCC and PVTT, with a more apparent OS benefit signal in Vp3– 4 disease and no clear increase in grade 3– 4 AEs. Keywords: hepatocellular carcinoma, portal vein tumor thrombosis, locoregional therapy, immune checkpoint inhibitor, prognosis

Source: Journal of Hepatocellular Carcinoma

DOI: 10.2147/jhc.s624104