Phase II Study of Selumetinib in Children and Young Adults With Tumors Harboring Activating Mitogen-Activated Protein Kinase Pathway Genetic Alterations: Arm E of the NCI-COG Pediatric MATCH Trial.
Olive S EcksteinCarl E AllenP Mickey WilliamsSinchita Roy-ChowdhuriDavid R PattonBrent CoffeyJoel M ReidJin PiaoLauren SaguiligTodd A AlonzoStacey L BergNilsa C RamirezAlok I JajuJoyce MhlangaElizabeth FoxDouglas S HawkinsMargaret M MooneyNaoko TakebeJames V TricoliKatherine A JanewayNita L SeibelDonald Williams ParsonsPublished in: Journal of clinical oncology : official journal of the American Society of Clinical Oncology (2022)
A national histology-agnostic molecular screening strategy was effective at identifying children and young adults eligible for treatment with selumetinib in the first Pediatric MATCH treatment arm to be completed. MEK inhibitors have demonstrated promising responses in some pediatric tumors (eg, low-grade glioma and plexiform neurofibroma). However, selumetinib in this cohort with treatment-refractory tumors harboring MAPK alterations demonstrated limited efficacy, indicating that pathway mutation status alone is insufficient to predict response to selumetinib monotherapy for pediatric cancers.