Multicenter phase 2 trial of pembrolizumab monotherapy for rare sarcomas

Sarcoma is a heterogeneous group of diseases with few treatment options. The multicenter phase 2 AcSé Pembrolizumab trial, at 24 centers in France, evaluated pembrolizumab monotherapy for rare sarcomas (incidence less than one case per one million people per year). At week 12, objective response rate was 6.2% (95% CI 2.3-13.0); all partial responses. The most common grade 3 or 4 adverse events were anemia , transaminase increases, and dyspnoe. None of the deaths due to adverse events were determined to be related to treatment. The authors conclude that pembrolizumab had activity and manageable toxicity in some rare sarcoma histotypes.