Predictive value of tumor mutational burden for pembrolizumab efficacy for advanced solid tumors

The accumulation of neoantigens increases the likelihood that tumor cells will be recognized by infiltrating immune cells. Somatic tumor mutational burden (TMB) may result in neoantigen formation. Agents that promote T-cell activation may be particularly effective in cancer patients with high TMB. A pan-tumor retrospective analysis of patients with previously treated advanced solid tumors in twelve studies found that TMB ≥ 175 mutations per exome was associated with improved efficacy of pembrolizumab monotherapy and improved outcomes with pembrolizumab versus chemotherapy. The analysis found that the predictive value of TMB was independent of tumor type, PD-L1 expression, or MSI status.