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Efficacy of Nivolumab plus Ipilimumab in Advanced Hepatocellular Carcinoma with and without High-Risk Features: An International Multicenter Study - PubMed

3 hours ago
  • #hepatocellular carcinoma
  • #real-world study
  • #immunotherapy
  • Nivolumab plus ipilimumab (Nivo/Ipi) showed efficacy in advanced hepatocellular carcinoma (HCC) in clinical trials, but high-risk patients (e.g., ≥50% liver involvement, Vp4 PVTT, bile duct invasion) were excluded.
  • This real-world international study of 145 advanced HCC patients (44 high-risk, 101 non-high-risk) found significantly worse outcomes in high-risk patients: lower objective response rate (12.8% vs. 34.3%), shorter progression-free survival (1.2 vs. 2.9 months), and overall survival (3.6 vs. 16.5 months).
  • Among high-risk features, ≥50% liver involvement was strongly associated with inferior treatment response and survival, while Vp4 portal vein tumor thrombosis and bile duct invasion alone did not significantly affect outcomes.
  • High-risk patients experienced more adverse events, especially hyperbilirubinemia, and multivariable analysis identified ≥50% liver involvement and prior immune-checkpoint inhibitor exposure as independent predictors of shorter survival.