Journal of neuro-oncology · 2026
MEK inhibitors in NF1-associated high-grade glioma: partial responses and outcomes from a single-institution cohort and literature review.
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Yu Ming-Chun, Hsi Aileen H, Adnan Aqsa, Knight Stephanie R, McCutcheon Ian E, Slopis John M, Kamiya-Matsuoka Carlos
Аннотация
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.
RESULTS: Nine patients were included. Median age at HGG onset was 34 years (range, 22-58), and median Karnofsky Performance Status at MEKi initiation was 60 (range, 50-90). Diagnoses were high-grade astrocytoma with piloid features (n = 6), IDH-wildtype glioblastoma (n = 2), and IDH-wildtype HGG/not otherwise specified (n = 1). Most patients received MEKi after prior local and/or systemic therapy. Among three patients treated with mirdametinib, two evaluable patients achieved partial response by Response Assessment in Neuro-Oncology criteria. Six patients received selumetinib for 1-7 cycles, and one patient received dabrafenib/trametinib for 2 cycles. Stable disease was the best response among the remaining evaluable patients. Median PFS and OS from MEKi initiation were 2.9 and 9.4 months, respectively.
CONCLUSION: Radiographic partial responses were observed in two patients with NF1-associated HGG treated with mirdametinib, but durability of benefit was limited. The best response with selumetinib and dabrafenib/trametinib was stable disease in evaluable patients. Larger prospective studies are needed to define the role of MEK inhibition in this population.
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.
RESULTS: Nine patients were included. Median age at HGG onset was 34 years (range, 22-58), and median Karnofsky Performance Status at MEKi initiation was 60 (range, 50-90). Diagnoses were high-grade astrocytoma with piloid features (n = 6), IDH-wildtype glioblastoma (n = 2), and IDH-wildtype HGG/not otherwise specified (n = 1). Most patients received MEKi after prior local and/or systemic therapy. Among three patients treated with mirdametinib, two evaluable patients achieved partial response by Response Assessment in Neuro-Oncology criteria. Six patients received selumetinib for 1-7 cycles, and one patient received dabrafenib/trametinib for 2 cycles. Stable disease was the best response among the remaining evaluable patients. Median PFS and OS from MEKi initiation were 2.9 and 9.4 months, respectively.
CONCLUSION: Radiographic partial responses were observed in two patients with NF1-associated HGG treated with mirdametinib, but durability of benefit was limited. The best response with selumetinib and dabrafenib/trametinib was stable disease in evaluable patients. Larger prospective studies are needed to define the role of MEK inhibition in this population.