Published online Aug 14, 2020. doi: 10.3748/wjg.v26.i30.4465
Peer-review started: March 14, 2020
First decision: April 12, 2020
Revised: April 25, 2020
Accepted: July 4, 2020
Article in press: July 4, 2020
Published online: August 14, 2020
Lenvatinib has become an indispensable part of treatment regimens for patients with advanced hepatocellular carcinoma (aHCC). Several recent real-world studies appear to have confirmed this; however, there are etiological differences. This necessitates further real-world studies of lenvatinib across diverse populations, such as in China.
To investigate the efficacy and safety of lenvatinib in a Chinese HCC patient population under real-world conditions.
This is a retrospective and multiregional study involving patients with aHCC receiving lenvatinib monotherapy. Efficacy was assessed using the Response Evaluation Criteria in Solid Tumors version 1.1. Baseline characteristics and adverse events (AEs) were recorded throughout the entire study.
In total, 54 HCC patients treated with lenvatinib monotherapy were included for final analysis. The objective response rate was 22% (n = 12) with a progression-free survival (PFS) of 168 d; however, AEs occurred in 92.8% of patients. Multivariate analysis showed that the Barcelona Clinic Liver Cancer stage [hazard ratio (HR) 0.465; 95%CI: 0.23-0.93; P = 0.031], portal vein tumor thrombus (HR 0.38; 95%CI: 0.15-0.94; P = 0.037) and Child-Pugh classifications (HR 0.468; 95%CI: 0.22-0.97; P = 0.042) were significant factors affecting PFS. The sensitivity (56.7%) and specificity (83.3%) of decreasing serum biomarkers including alpha-fetoprotein were calculated in order to predict tumor size reduction. Gene sequencing also provided insights into potential gene mutation signatures related to the effect of lenvatinib.
Our findings confirm previous evidence from the phase III REFLECT study. The majority of patients in this Chinese sample were suffering from concomitant hepatitis B virus-related HCC. However, further analysis suggested that baseline characteristics, changes in serum biomarkers and gene sequencing may hold the key for predicting lenvatinib responses. Further large-scale prospective studies that incorporate more basic medical science measures should be conducted.
Core tip: This is a real-world study of advanced hepatocellular carcinoma patients treated with lenvatinib monotherapy in China. The majority of patients in this study presented with hepatitis B virus infection. Our analysis of the safety and efficacy of this intervention confirms previous evidence from the phase III REFLECT study. A multivariate analysis of participant characteristics with changes in serum biomarkers and gene sequencing provides a more comprehensive understanding of lenvatinib responses. Although based on a small sample, this new knowledge has clinical implications and necessitates further research.