Nivolumab for Sorafenib-Naïve, Sorafenib-Experienced Patients With Advanced HCC

A 5-year follow-up analysis of the CheckMate 040 trial sought to determine the safety and tolerability of nivolumab for patients with advanced hepatocellular carcinoma (aHCC) who are sorafenib-naive and sorafenib-experienced.

The study was led by Anthony B. El-Khoueiry, MD, and published in Annals of Oncology.

Researchers noted that patients with aHCC often experience high mortality and a poor prognosis. The CheckMate 040 trial reported that nivolumab monotherapy benefits patients who have an acceptable safety profile.

For the trial, patients received nivolumab monotherapy in a 0.1- to 10.0-mg/kg dose or a 3-mg/kg dose every 2 weeks until reaching either disease progression or unacceptable toxicity. The study’s primary end points included safety, tolerability, and objective response rate (ORR).

Dr. El-Khoueiry and colleagues evaluated the data of 80 sorafenib-naïve patients and 154 sorafenib-experienced patients participating in the trial.

According to a blinded, independent, central review, the ORR was 20% (95% CI, 12-30) and 14% (95% CI, 9-21) for the sorafenib-naive and sorafenib-experienced patients, respectively.

For sorafenib-naïve patients, median overall survival (OS) was 26.6 months (95% CI, 16.6-30.6 months), the 3-year OS rate was 28%, and the 5-year OS rate was 14%. In the sorafenib-experienced group, median OS was 15.1 months (95% CI, 13.0-18.2 months), the 3-year OS rate was 20%, and the 5-year OS rate was 12%.

Researchers found no new safety signals, with 33% of patients in the sorafenib-naïve cohort and 21% in the sorafenib-experienced group reporting grade 3 or 4 treatment-related adverse events.

Baseline PD-L1 expression ≥1% related to an increased ORR and OS when compared with PD-L1 expression ≤1%. Sorafenib-naïve patients with OS ≥3 years demonstrated an increased baseline CD8 T-cell density compared with patients with an OS ≤1 year.

“With 5 years of follow-up, nivolumab monotherapy continued to provide durable clinical benefit with manageable safety in sorafenib-naive and sorafenib-experienced patients with aHCC,” Dr. El-Khoueiry and colleagues concluded.