Efficacy of nivolumab in pediatric cancers with high mutation burden and mismatch-repair deficiency.
Anirban DasUri TaboriLauren C Sambira NahumNatalie B CollinsRebecca J DeyellRina DvirCecile Faure ConterTimothy Eg HassallJane E MinturnMelissa EdwardsElissa BrookesVanessa BianchiAdrian B LevineSimone Cardozo StoneSumedha SudhamanSantiago Sánchez-RamírezAyse Bahar ErcanLucie K StengsJiil ChungLogine NegmGad A GetzYosef E MaruvkaBirgit Betina Ertl-WagnerPamela S OhashiTrevor J PughCynthia HawkinsEric BouffetDaniel A MorgensternPublished in: Clinical cancer research : an official journal of the American Association for Cancer Research (2023)
Nivolumab resulted in durable responses and prolonged survival for the first time in a pediatric trial of refractory hypermutated cancers including malignant gliomas. Novel biomarkers identified here need to be translated rapidly to clinical care to identify children who can benefit from checkpoint-inhibitors, including for upfront management of their cancers.