Efficacy and Safety of Durvalumab Plus Tremelimumab in Hepatocellular Carcinoma Patients With Portal Vein Thrombosis and High Tumor Burden: A Multicenter Retrospective Analysis

Aims This study aimed to evaluate the therapeutic efficacy of durvalumab and tremelimumab (Dur/Tre) in patients with hepatocellular carcinoma (HCC) who had a tumor thrombus in the main portal vein trunk (Vp4) or high tumor burden (HTB). Methods A total of 309 patients with BCLC stage B or C HCC who received Dur/Tre between March 2023 and October 2024 were included. HTB was defined as the presence of at least one of the following radiological findings: ≥ 50% liver involvement by HCC, bile duct invasion, or the presence of Vp4. Results Both the patients with Vp4 and HTB‐positive group had significantly higher proportions of BCLC stage C disease ( p = 0.01 and 0.007, respectively) and serum DCP levels ≥ 100 mAU/mL ( p = 0.03 and < 0.001, respectively), and significantly higher neutrophil‐to‐lymphocyte ratio ( p = 0.04 and p = 0.004, respectively) compared to their respective counterparts. While the objective response rate did not significantly differ between the HTB‐positive and HTB‐negative groups (21.6% vs. 16.2%, p = 0.5), it was significantly higher in patients with Vp4 than in those without (42.9% vs. 15.6%, p = 0.02). There were no significant differences in progression‐free survival or overall survival (OS) between patients with and without Vp4 ( p = 0.1 and 0.3, respectively) and nor between the HTB‐positive and HTB‐negative groups (both p = 0.3). Among patients with both Vp4 and HTB, responders had longer OS than non‐responders. Conclusions Dur/Tre may be a viable treatment option for patients with Vp4 and HTB.