Sorafenib is one of the routinely assigned treatments for advanced hepatocellular carcinoma (HCC). But most patients with advanced HCC don’t respond to curative therapies, and thus, new treatments are required. This study aims to assess the safety and efficacy of nivolumab plus ipilimumab, a potential anticancer combination therapy, in patients with advanced HCC.

This multicenter, multicohort, open-label, phase 1/2 trial included a total of 148 participants (120 male) with advanced HCC previously treated with sorafenib. The patients were randomly assigned in a 1:1:1 ratio to receive nivolumab plus ipilimumab in three different dose combinations.  The primary outcomes of the study were safety, tolerability, and objective response rate.

During the median follow-up of 30.7 months, the investigator-assessed objective response rate was 32%, 27%, and 29% in three groups of varying dose concentrations. The median duration of response was not reached in one group and was 15.2 and 21.7 months in the other two groups. Treatment-related adverse events of any grade were reported in 71-94% of participants in all the groups combined.

The research concluded that nivolumab plus ipilimumab combination therapy had a manageable safety profile with durable responses and a positive objective response rate.