Real-world efficacy and prognostic factors of lenvatinib plus PD-1 inhibitors in 378 unresectable hepatocellular carcinoma patients

Abstract

Introduction Combining lenvatinib with a programmed cell death protein-1 (PD-1) inhibitor has been explored for the treatment of unresectable hepatocellular carcinoma (uHCC). This study aimed to investigate the real-world efficacy and prognostic factors of lenvatinib plus PD-1 inhibitor in large cohorts of Asia uHCC patients. Methods Patients with uHCC treated with lenvatinib and PD-1 inhibitors were included. The primary endpoints were overall survival (OS) and progression-free survival (PFS), and the secondary endpoints were the response rate (ORR) and adverse events (AEs). Prognostic factors of survival were also analysed. Results A total of 378 uHCC patients from two medical centres in China were included retrospectively. The median age was 55 years, and 86.5% of patients were male. Hepatitis B virus (HBV) infection (89.9%) was the dominant aetiology. The median OS was 17.8 (95% confidence intervals (CIs) 14.0-21.6) months. The median PFS was 6.9 (95% CI 6.0-7.9) months. The best objective response rate (ORR) and disease control rate (DCR) were 19.6% and 73.5%, respectively. In multivariate analysis, Child-Pugh score, Barcelona Clinic Liver Cancer stage, Eastern Cooperative Oncology Group performance status score, involved organs, tumour burden score, and combination with local therapy were independent prognostic factors for OS. 100% and 57.9% patients experienced all grade and grade 3/4 treatment-emergent AEs. Conclusion This real-world study of lenvatinib plus PD-1 inhibitors demonstrated long survival and considerable ORRs and DCRs for uHCC patients in China. The tolerability of combination therapy was acceptable but still be monitored closely.