Anti–programmed cell death protein 1 and anti–cytotoxic T-lymphocyte–associated protein 4 antibodies have shown activity in hepatocellular carcinoma. Based on these earlier findings, researchers sought to examine the safety, efficacy, and tolerability of perioperative treatment with nivolumab or nivolumab plus ipilimumab in resectable hepatocellular carcinoma. Findings from a small phase II trial were presented by Kaseb et al at the 2019 Gastrointestinal Cancers Symposium (Abstract 185).
A total of 9 patients were randomly assigned to receive either nivolumab (arm A) or nivolumab plus ipilimumab (arm B). All patients enrolled were treated with 240 mg of nivolumab every 2 weeks for a total of 6 weeks; patients in arm B were also treated concurrently with ipilimumab at 1 mg/kg every 6 weeks. Surgical resection was performed within 4 weeks after the last cycle of therapy, and patients continued adjuvant treatment for up to 2 years after resection.
The primary endpoint was the safety and tolerability of nivolumab or nivolumab plus ipilimumab. Secondary endpoints were overall response rate, complete response rate, and time to progression.
Eight patients were evaluable at the time of first interim analysis (5 in arm A and 3 in arm B). Pathologic complete response was observed in 3 (37.5%) of 8 patients—2 patients treated with nivolumab monotherapy and 1 patient treated with the immunotherapy combination.
A total of 3 patients experienced grade 3 or higher toxicity—which did not affect their resection—and no grade 4 or higher toxicity was reported. The pathologic complete response in the patient treated with nivolumab plus ipilimumab showed that clinical response correlated with an increase in CD8-positive T-cell infiltration—notably, an increase in two effector T-cell clusters.
The investigators concluded, “Treatment was deemed safe, and surgical resection was not delayed. The study is ongoing, and results may contribute to a paradigm shift in the perioperative treatment of hepatocellular carcinoma.”
Disclosure: The study authors' full disclosures can be found at coi.asco.org.
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