Characteristics of cabozantinib treatment in advanced hepatocellular carcinoma

Abstract Background and Aim Cabozantinib is a molecular targeted agent (MTA) used for treatment of advanced hepatocellular carcinoma (HCC). Although its superiority over placebo has been proven, its effectiveness and risk factors in real‐world practice are needed to be elucidated. Methods This study retrospectively enrolled 54 advanced HCC patients, who were treated with cabozantinib. The effectiveness of cabozantinib, adverse events (AE) and risk factors for survival was analysed. Results Majority of the patients (88.9%) were treated with two or more MTAs before starting cabozantinib and atezolizumab plus bevacizumab was the most prevalent MTA used (59.3%). The median overall survival and progression‐free survival (PFS) were 6.9 and 4.4 months, respectively. The objective response rate and disease control rate were 3.7% and 40.7%, respectively. Grade 3/4 AE occurred in 37.0% of the patients; however, unpredictable AE was not observed. Multivariate analysis revealed that high neutrophil–lymphocyte ratio (NLR, >4) was a risk factor for survival (hazard ratio for death, 2.35; 95% confidence interval [CI], 1.41–4.82; p = 0.020). Moreover, the occurrence of Grade 3/4 AE was a negative risk factor for both survival (hazard ratio for death, 0.36; 95% CI, 0.16–0.83; p = 0.016) and PFS (hazard ratio for disease progression or death, 0.33; 95% CI, 0.15–0.73; p = 0.006). Neither preceding therapy with atezolizumab/bevacizumab nor a reduced starting dose correlated with patient survival. Conclusions Cabozantinib can be used safely in real‐world practice. The study identified high NLR as a positive risk factor and the occurrence of Grade 3/4 AE as a negative risk factor for survival.