Early Trial Finds Immunotherapy Effective in Liver Cancer


Results from a phase I/II study suggest that the immunotherapy nivolumab is safe and effective in advanced hepatocellular carcinoma (HCC).

Results from a phase I/II study suggest that the PD-1 inhibitor nivolumab is safe and effective in advanced hepatocellular carcinoma (HCC). Data from the trial (abstract LBA101) were presented in a press conference at the 2015 American Society of Clinical Oncology (ASCO) Annual Meeting, held May 29 to June 2, in Chicago.

“We are encouraged to see that nivolumab was safe overall, and the response rate as well as preliminary survival data look quite promising,” said study author Anthony B. El-Khoueiry, MD, associate professor of clinical medicine at the University of Southern California Norris Comprehensive Cancer Center in Los Angeles, in a press release. “While we have to verify this early signal in larger studies, this is one of the first signs that immunotherapy with immune checkpoint inhibitors will have a role in the treatment of liver cancer.”

The study included 42 evaluable patients with advanced HCC whose disease progressed on sorafenib, or who either refused or were intolerant of the drug. Patients enrolled in the study had a Child-Pugh (CP) score ≤ B7. The tyrosine kinase inhibitor sorafenib is the only FDA-approved systemic treatment for advanced HCC, but only 2% of patients have an objective tumor response to the drug.

In the phase I part of the study, 8 of 42 patients (19%) responded to treatment with nivolumab with a tumor reduction greater than 30%, with 2 patients experiencing complete remissions. Nivolumab was administered intravenously every 2 weeks at a dose of 0.1–10 mg/kg for up to 2 years. No maximum tolerated dose was found. Responses have proved durable, surpassing 12 months in 4 of the 8 responding patients. The rate of overall survival was 62% at 12 months.

Drug-related adverse events occurred in 71% of patients. Common events (≥ 10% of patients) included rash (17%), and increases in aspartate aminotransferase (17%), alanine aminotransferase (15%), lipase (15%), and amylase (12%). The most common (≥ 5% of patients) grade 3/4 adverse events were increases in aspartate aminotransferase (12%), alanine aminotransferase (10%), and lipase (5%). Two patients discontinued therapy due to drug-related side effects.

“While these results are preliminary and limited to a small number of patients, they remain exciting and provide strong justification for more studies of nivolumab and other immunotherapy approaches for patients with advanced liver cancer,” said El-Khoueiry.

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