In a small phase 1/2 safety study, 19% of previously treated, advanced HCC patients responded to of the PD-1 inhibitor nivolumab.1

Anthony B. El-Khoueiry from the University of Southern California Norris Comprehensive Cancer Center in Los Angeles, noted that patients with hepatocellular carcinoma (HCC) who have advanced on sorafenib have very poor prognosis and few additional treatment options; sorafenib is the standard of care.

Further, overexpression of the immune checkpoint PD-L1 in HCC is associated with aggressive tumor growth and recurrence after surgery, supporting a rationale for nivolumab treatment.2

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The primary outcome measure of the study was safety; patients received doses of nivolumab ranging from 0.1 to 10.0 mg/kg for up to 2 years.

Dose escalation was done in parallel groups based on whether patients were positive for hepatitis B (HBV; 11 patients) or hepatitis C (HCV; 12 patients) or were free of either virus (24 patients).

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To be enrolled in the study, patients had to have a Child-Pugh score 7 or less and histologically confirmed HCC; all were ineligible for curative therapy. Enrolled patients had progressive disease either on sorafenib or were intolerant of or refused sorafenib treatment. Objective responses (mRECIST criteria) were a secondary outcome measure.2

Of enrolled patients, 77% had prior sorafenib treatment. Dr. El-Khoueiry said that 17 patients remained on nivolumab treatment at present and 30 had discontinued, only 2 due to an adverse reaction. Two others discontinued because they achieved a complete response.