The U.S. Food and Drug Administration (FDA) has approved nivolumab plus ipilimumab for first-line therapy in adult patients with unresectable or metastatic hepatocellular carcinoma (HCC).
HCC is a rare carcinoma of the liver characterized by nodules occurring anywhere within the liver, composed of neoplastic epithelial cells with hepatocellular differentiation. The vast majority of tumors are associated with chronic liver disease or exposure to a variety of exogenous agents. Common symptoms include right upper quadrant abdominal pain, weight loss, hepatosplenomegaly, jaundice, and ascites.
The approval was largely based on the CheckMate-9DW clinical trial, a phase 3, randomized, open-label study that evaluated the combination of nivolumab plus ipilimumab compared to tyrosine kinase monotherapy in patients with HCC. Nivolumab is a monoclonal antibody that targets the PD1 receptor and obstructs the activation of T-cells. Ipilimumab is a CTLA-4 monoclonal antibody that also targets T-cell activation. The primary endpoint of this trial was overall survival with key secondary endpoints of objective response rate and time to symptom deterioration.
The mean overall survival in the combination therapy group was 23.7 months versus 20.6 months in the monotherapy group, resulting in a 21% reduction in risk of death. Overall survival rate at three years was 38% in the combination group versus 24% in the monotherapy group. Overall response rates were also significant in the combination group at 36.1% compared 13.2% in the monotherapy group. Responses were also longer in the combination group with a median duration of response of 30.4 months versus 12.9 months in the monotherapy group. Additionally, no new safety signals were reported.
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To learn more about HCC and other rare cancers, visit https://checkrare.com/diseases/cancers/