Journal of neuro-oncology
MEK inhibitors in NF1-associated high-grade glioma: partial responses and outcomes from a single-institution cohort and literature review
By Yu MC, Hsi AH, Adnan A and 4 others.
Abstract
Purpose
NF1-associated high-grade gliomas (HGGs) are rare, biologically heterogeneous tumors with poor outcomes. Although MEK inhibitors (MEKis) are established options for selected NF1-associated low-grade gliomas, their role in NF1-associated HGG remains undefined. We describe clinical and radiographic outcomes with MEKis, including mirdametinib.
Methods
We retrospectively reviewed adult patients with NF1-associated HGG treated at The University of Texas MD Anderson Cancer Center who initiated selumetinib, mirdametinib, or dabrafenib plus trametinib from December 1, 2019, to January 31, 2026. Outcomes included radiographic response and Kaplan-Meier estimates of progression-free survival (PFS) and overall survival (OS) from MEKi initiation. Progression was defined as the earliest documented radiographic or clinical evidence of progression.