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← Primary Liver Cancer

HEALTH PROFESSIONAL · SOURCE READING

Nivolumab

Source: Primary Liver Cancer Treatment (PDQ®)–Health Professional Version, National Cancer Institute.

Source updated: April 17, 2025 · Captured 2026-09-09.

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Context: Treatment of Locally Advanced or Metastatic Primary Liver Cancer / Treatment Options for Locally Advanced or Metastatic Primary Liver Cancer / Second-line systemic therapy / Combination immunotherapy and immunotherapy alone

Evidence (nivolumab):

A phase I/II, open-label, single-arm, dose-escalation and dose-expansion trial (CheckMate 040 [NCT01658878]) included 262 patients with advanced HCC and well-compensated liver function. Of those patients, 48 were enrolled in the dose-escalation phase and 214 patients were enrolled in the dose-expansion phase with nivolumab 3 mg/kg. Patients were treated with nivolumab every 2 weeks.[28] Cohorts included patients with active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection, uninfected patients with sorafenib-naïve disease, and uninfected patients with sorafenib-refractory disease.

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A phase I/II, open-label, single-arm, dose-escalation and dose-expansion trial (CheckMate 040 [NCT01658878]) included 262 patients with advanced HCC and well-compensated liver function. Of those patients, 48 were enrolled in the dose-escalation phase and 214 patients were enrolled in the dose-expansion phase with nivolumab 3 mg/kg. Patients were treated with nivolumab every 2 weeks.[28] Cohorts included patients with active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection, uninfected patients with sorafenib-naïve disease, and uninfected patients with sorafenib-refractory disease.

The total overall objective response rate in the dose-expansion phase was 20% (95% CI, 15%–26%) with three complete responses. Results were similar in untreated, refractory, and HBV/HCV-infected patients.[28][Level of evidence B4]

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However, based on postmarketing requirements showing lack of confirmatory benefit, the indication for nivolumab monotherapy in the second-line setting was withdrawn in 2021.

Studies cited in this source section

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Preserved source evidence · Not medical advice