Excellent Response to Anti-PD-1 Therapy in a Patient with Hepatocellular Carcinoma Intolerant to Sorafenib.

BACKGROUND: Hepatocellular carcinoma (HCC) represents the most common primary carcinoma of the liver. Most patients present with advanced or metastatic HCC at diagnosis and face a very limited prognosis. Systemic treatment with the tyrosine-kinase inhibitors sorafenib or lenvatinib is considered as the treatment of choice in these patients. In patients intolerant to tyrosine-kinase inhibitors, no standard therapy is approved so far, but several agents have demonstrated efficacy in clinical trials. As such, the checkpoint inhibitors nivolumab and pembrolizumab have been shown to induce tumor response and to prolong survival in patients with advanced HCC, both when intolerant to sorafenib or after failure of a first-line therapy with sorafenib. CASE REPORT: We here report the case of a patient with advanced HCC that demonstrated severe toxicity upon first-line treatment with sorafenib. Administration of sorafenib was discontinued and after careful consideration the patient started a second-line treatment with the PD-1 antibody nivolumab. Strikingly, this treatment led to a significant regression of tumor size and a drastic reduction of alpha-fetoprotein serum concentrations. At 17 months after initiation of treatment, the patient is still alive in excellent condition with sustained tumor response.
CONCLUSION: In summary, we report on a very rare case of a patient with HCC demonstrating an almost complete response to checkpoint inhibitor treatment.