Cabozantinib demonstrates favourable risk–benefit profile in pretreated advanced HCC

Use of the vascular endothelial growth factor (VEGF) inhibitor cabozantinib in the second- or third-line treatment of patients with advanced hepatocellular carcinoma (HCC) appears to confer survival benefits without raising new safety concerns, according to the results of a phase II trial.

In total, 34 patients (median age, 73.0 years; 91.2 percent male) received cabozantinib 60 mg orally once daily, among whom 20 had previously received sorafenib (median duration, 7.9 months). Most patients (76.5 percent) had received one prior systemic anticancer regimen, while some (23.5 percent) received two.

The primary endpoint of progression-free survival (PFS) at week 24 was achieved in 40.1 percent (90 percent confidence interval [CI], 24.8–55.0) overall, 59.8 percent (90 percent CI, 36.1–77.2) in the prior sorafenib cohort, and 16.7 percent (90 percent CI, 4.0–36.8) in the sorafenib-naïve cohort.

Median PFS in the corresponding cohorts was 5.6, 7.4, and 3.6 months.

Six-month overall survival rate was 91.1 percent in the entire population, 100.0 in the prior sorafenib cohort, and 78.6 percent in the sorafenib-naïve cohort. The respective disease control rates were 76.5 percent, 85.0 percent, and 64.3 percent. The objective response rates were 0.0 percent both in prior sorafenib and naïve cohorts.

In terms of safety, all patients required dose modifications due to adverse events (AEs). The most frequently reported AEs were palmar–plantar erythrodysesthesia syndrome and diarrhoea. AEs led to treatment discontinuation in three patients.