Systemic Treatment Patterns and Clinical Outcomes in Hepatocellular Carcinoma: A Multicenter Real-World Study from...

Systemic therapy for hepatocellular carcinoma (HCC) has evolved rapidly with the introduction of tyrosine kinase inhibitors (TKI), immune checkpoint inhibitors (ICIs), anti-vascular endothelial growth factor (anti-VEGF) antibodies, and their combinations. This multicenter study aimed to characterize real-world systemic treatment patterns and clinical outcomes in treatment-naïve patients with HCC in China.

Patients initiating systemic therapy for HCC between 2017 and 2023 across five centers were retrospectively enrolled. Four systemic treatment categories were analyzed: TKI monotherapy, ICIs monotherapy, TKI plus ICIs, and anti-VEGF plus ICIs. Baseline clinical characteristics, treatment patterns across lines, survival outcomes, tumor response, and adverse events (AEs) were analyzed.

A total of 4,250 patients were included (median age, 53 years), predominantly male (88.7%) with hepatitis B virus infection (86.3%); 81.3% had Barcelona Clinic Liver Cancer stage C disease. In the first-line setting, 92.3% of patients received systemic therapy combined with transarterial therapy. Median overall survival (OS) was 20.7 months (95% CI, 19.1-22.3), and median progression-free survival (PFS) was 9.7 months (95% CI, 8.2-11.2). Baseline characteristics differed across systemic treatment groups, including demographic and disease characteristics and treatment patterns involving transarterial therapy, and were accounted for using inverse probability of treatment weighting and multivariable Cox regression. Median OS was 17.0, 21.4, 21.6, and 22.4 months in the TKI monotherapy, ICIs monotherapy, TKI plus ICIs, and anti-VEGF plus ICIs groups, respectively. Grade ≥3 AEs occurred in 28.3% of patients.

In this multicenter cohort, ICIs-based systemic regimens combined with transarterial therapy were frequently applied in patients with HCC and associated with favorable survival outcomes and acceptable safety profiles. These findings provide contemporary real-world insights into systemic treatment patterns and outcomes.

The therapeutic landscape of hepatocellular carcinoma (HCC) has evolved rapidly, whereas treatment patterns in routine clinical practice may differ substantially from those represented in randomized clinical trials, particularly in regions with a high burden of hepatitis B-related disease. This large multicenter real-world study delineates contemporary systemic treatment strategies in China and describes clinical outcomes associated with the use of immune checkpoint inhibitor-containing regimens, particularly when integrated with transarterial therapy, in routine clinical practice. These findings provide additional real-world context regarding multidisciplinary treatment approaches for HCC and may help inform future prospective evaluation of systemic and locoregional treatment integration. Although limited by its observational design, this study generates hypotheses for future prospective studies evaluating multimodal treatment strategies and patient selection for immunotherapy-based approaches in HCC.