Pediatric glioma patients typically respond poorly to immune checkpoint inhibitors (ICI) due to an immune suppressive environment, low tumor mutational burden (TMB), low MHC expression, and heterogeneity of mismatch repair deficient (MMRD) cells within glioma tumors. However, a subset of MMRD-driven hypermutated pediatric glioma patients may benefit from ICI therapy. In this open-label, single-arm pilot study, Das and colleagues assessed the safety and efficacy of the anti-PD-1 immunotherapeutic nivolumab in pediatric patients with relapsed/refractory cancers with elevated TMB and/or MMRD. The authors found that nivolumab was well tolerated, with most patients experiencing grade ≤ 2 treatment-related adverse events (TRAE) and only one patient discontinuing treatment due to grade 3 pancreatitis. A delayed immune response led to an initial overall response rate (ORR) of 20%, however the ORR increased to 50% after the trial ended, with 30% of patients achieving complete response and 20% of patients demonstrating partial response. At a...

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