Retrospective study links combined HAIC and systemic therapy to outcomes in advanced liver cancer

A retrospective study of 331 people who were initially diagnosed with hepatocellular carcinoma (HCC) and major portal vein tumor thrombosis found that hepatic arterial infusion chemotherapy (HAIC) combined with immune checkpoint inhibitors and/or targeted therapy was associated with an objective response rate of 61.3%. Patients had Vp3/4 portal vein tumor thrombosis, and the study reported a median progression-free survival of 10.0 months and median overall survival of 15.0 months. Because the study did not include a randomized comparison group, these results describe outcomes observed under the combined treatment strategy rather than demonstrating that it was superior to systemic therapy, locoregional treatment alone, or another standard approach.

The researchers enrolled patients treated between December 2018 and April 2024 and reviewed demographic and laboratory data, treatment allocation, tumor response, survival, and adverse events. The cohort was restricted to initially diagnosed HCC with Vp3/4 portal vein tumor thrombosis, so the findings do not directly describe recurrent disease or patients with less extensive portal vein involvement. Tumor response was assessed using RECIST 1.1 criteria. The retrospective, single-arm design and selected treatment cohort limit the conclusions that can be drawn about comparative effectiveness, and the reported survival estimates cannot separate the effects of HAIC, immune checkpoint inhibitors, targeted therapy, and their combination.

Multivariable Cox regression analyses identified clinical and laboratory characteristics associated with survival. Age, aspartate aminotransferase (AST), alkaline phosphatase (ALP), C-reactive protein (CRP), Child-Pugh grade, and metabolic comorbidity were associated with overall survival. For progression-free survival, the associated factors were AST, ALP, CRP, extrahepatic metastasis, and metabolic comorbidity. The authors also described a set of favorable prognostic characteristics: age of 62.5 years or younger, Child-Pugh grade A, AST of 63.5 U/L or lower, ALP of 176.5 U/L or lower, CRP of 17.8 mg/L or lower, no extrahepatic metastasis, and coexistence of metabolic comorbidity. The investigators characterized these findings as hypothesis-generating observations that require external and prospective confirmation. They are prognostic associations from the same cohort, not validated criteria for treatment selection, and the analysis did not establish that the factors predict differential benefit from the combined regimen.

Safety outcomes were assessed alongside tumor response and survival. The most common grade 3–4 adverse events were thrombocytopenia, elevated total bilirubin, fever, upper gastrointestinal bleeding, and elevated transaminases, each occurring in 7.3–7.6% of patients. Ascites occurred in 5.4%, and no treatment-related deaths were reported. Without a comparison group, the study could not determine whether these toxicities were more frequent or less frequent than those associated with alternative treatment strategies. The findings instead document the adverse events observed among the 331 treated patients in this specific advanced-HCC population.

Overall, the study provides preliminary evidence of antitumor activity and survival outcomes for HAIC combined with immune checkpoint inhibitors and/or targeted therapy in initially diagnosed HCC with Vp3/4 portal vein tumor thrombosis. The cohort’s median progression-free survival of 10.0 months, with a 95% confidence interval of 8.7–14.3 months, and median overall survival of 15.0 months, with a 95% confidence interval of 13.2–16.8 months, indicate that these were the central outcomes observed under the regimen. The findings remain limited by the retrospective design, absence of a control group, and restriction to a single advanced-disease group. Controlled and prospective studies are needed to establish comparative effectiveness and determine whether the reported prognostic associations hold in other populations.

Subject of Research: Outcomes of combined HAIC and systemic treatment for hepatocellular carcinoma with major portal vein tumor thrombosis

Article Title: Revisiting HAIC and systemic treatment for initially diagnosed HCC with major portal vein (Vp3/4) tumor thrombosis

Article References: Chen, M., Zhang, L., Liu, C., Yuan, J., Zhang, J., Xu, X., Ge, N., Zhang, L., & Chen, R. (2026). Revisiting HAIC and systemic treatment for initially diagnosed HCC with major portal vein (Vp3/4) tumor thrombosis. BMC Cancer. https://doi.org/10.1186/s12885-026-16777-7

Image Credits: AI Generated

DOI: 10.1186/s12885-026-16777-7

Keywords: hepatocellular carcinoma, portal vein tumor thrombosis, hepatic arterial infusion chemotherapy, immune checkpoint inhibitors, targeted therapy, retrospective cohort, objective response rate, progression-free survival, overall survival, treatment safety, Revisiting, HAIC