The following is a summary of “Pembrolizumab Versus Placebo as Second-Line Therapy in Patients From Asia With Advanced Hepatocellular Carcinoma: A Randomized, Double-Blind, Phase III Trial,” published in the March 2023 issue of Oncology by Qin, et al.

For a study, researchers sought to assess the effectiveness and safety of pembrolizumab in patients from Asia who had previously received treatment for advanced hepatocellular carcinoma (HCC).

In phase III double-blind trial, 453 patients with advanced HCC and progression during or after treatment with sorafenib or oxaliplatin-based chemotherapy were randomly assigned in a 2:1 ratio to receive either pembrolizumab (200mg) or placebo once every three weeks for ≤35 cycles along with best supportive care. The primary endpoint was overall survival (OS) (one-sided significance threshold, P = .0193 [final analysis]). The secondary endpoints were progression-free survival (PFS) and objective response rate (ORR) (one-sided significance threshold, P = .0134 and .0091, respectively [second interim analysis]; RECIST version 1.1, by blinded independent central review).

The results showed that the median overall survival was longer in the pembrolizumab group (14.6 months) than in the placebo group (13.0 months). The death hazard ratio (HR) for death was 0.79 (95% CI, 0.63 to 0.99; P = .0180). Median PFS was also longer in the pembrolizumab group (2.6 months) than in the placebo group (2.3 months). The HR for progression or death was 0.74 (95% CI, 0.60 to 0.92; P = .0032). The objective response rate was higher in the pembrolizumab group (12.7%) than in the placebo group (1.3%) with a P-value of less than 0.0001. Treatment-related adverse events occurred in 66.9% of patients (grade 3, 12.0%; grade 4, 1.3%; grade 5, 1.0%) in the pembrolizumab group and 49.7% of patients (grade 3, 5.9%; grade 4, 0%; grade 5, 0%) in the placebo group.

In conclusion, in patients from Asia with previously treated advanced HCC, pembrolizumab was found to significantly prolong overall survival and PFS and had a higher ORR when compared to placebo.

Reference: ascopubs.org/doi/full/10.1200/JCO.22.00620