Peripheral inflammatory indices and outcomes under PD-1/PD-L1–based regimens in HCC: an exploratory single-center retrospective analysis

Abstract Purpose Immune checkpoint inhibitors (ICIs) are the preferred treatment for advanced hepatocellular carcinoma (HCC). However, most patients do not respond to initial immunotherapy alone, even when combined with other therapies. Our study aimed to profile ICI-based therapies for HCC patients in real-world clinical practice and to identify factors associated with survival and treatment efficacy. Methods A retrospective cohort study was conducted to describe ICI-based therapies for HCC patients at our center, including combination modalities, effectiveness, and safety. Pre- and post-treatment indicators were collected to explore factors related to progression-free survival (PFS) and treatment efficacy in these patients. Results All 110 patients received therapy based on immune checkpoint inhibitors (ICIs) using three strategies, with the majority (60.9%) undergoing triple combination therapy. The overall progression-free survival (PFS) was 7.0 months, and the objective response rate (ORR) was 26.4%. Analysis of PFS identified several independent risk factors: a history of hepatitis B virus (HBV) infection (hazard ratio [HR] = 1.78), a platelet-to-lymphocyte ratio (PLR) greater than 276.9 (HR = 2.19), and an international normalized ratio (INR) greater than 1.2 (HR = 3.49). Conversely, the use of PD-1 inhibitors was associated with a favorable outcome (HR = 0.44). The combination of a history of HBV infection with HBeAg positivity and TNM stage (III + IV) effectively distinguished responders from non-responders. Additionally, neutrophil count and the neutrophil-to-lymphocyte ratio (NLR) were significantly elevated in non-responders and exhibited dynamic changes during treatment. Conclusion In HCC clinical practice, ICI-based systemic therapies are both effective and safe. Patient characteristics can serve as reliable predictors of progression-free survival (PFS) and treatment efficacy. Importantly, neutrophil count and the neutrophil-to-lymphocyte ratio (NLR) may also be used as potential biomarkers for efficacy conversion, rather than just as predictors.