Risk of developing malignant peripheral nerve sheath tumors in patients with neurofibromatosis 1 receiving MEK inhibitor treatment for plexiform neurofibromas

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ID: 323075
2026
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Abstract
Abstract Background Mitogen activating protein kinase kinase inhibitors (MEKi) provide clinical benefit to patients with symptomatic, inoperable plexiform neurofibromas (PNs) in Neurofibromatosis type 1 (NF1). Several cases of MPNST developing in patients while receiving MEKi for PN have been reported, raising the question whether MEKi therapy might increase this risk. We report the incidence of MPNST in those who have received MEKi, non-MEKi (e.g. mTOR inhibitors), and no PN-directed medical therapies and assess the risk of MPNST in patients who received MEKi. Methods Patients evaluated at the National Cancer Institute (NCI) with a clinical or genetic diagnosis of NF1 from 1/1/1998-12/31/2019 were included in this analysis (n = 296). Patients were categorized as MEKi (n = 29), non-MEKi (n = 91), both (n = 45), or no medical treatment (n = 131); only patients who received PN-directed medical treatment were included in the analysis of MPNST risk. Univariate evaluations of risk factors associated with MPNST were included in a multivariate model with treatment group as a time-dependent covariate. Results No difference in MPNST risk (HR:1.09, 95%CI: 0.30-4.01) between MEKi as compared to non-MEKi treatment was observed after adjusting for radiation exposure. Radiation exposure was associated with a higher risk of MPNST (HR 5.15, 95%CI: 1.15-23.04). Other patient characteristics, including NF1 inheritance pattern, were not associated with the risk of MPNST development in this cohort. Conclusion Our data does not demonstrate a significant difference in the risk of MPNST development between patients with NF1-PN who received MEKi and non-MEKi treatments. Radiation exposure was significantly associated with a higher risk of MPNST development.
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openalex_W7172011697 Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Hari Sankaran, Eva Dombi, Brittany Glassberg, Andrea Baldwin, Kara Heisey, Anne Dufek, Cecilia Tibery, Ana F. Best, Alice Chen, Brigitte C. Widemann, Andrea M. Gross
Journal Neuro-Oncology Advances
Year 2026
DOI
10.1093/noajnl/vdag201
URL
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