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Published on: October 10, 2025
Disease Stabilization with MEK Inhibitors in NF1-Associated Plexiform Neurofibromas: A Systematic Review and
Sai Sanikommu1, Alejandro N Santos2, Bashar M Dawoud2
1Department of Neurosurgery, Miller School of Medicine, University of Miami, 1501 NW 10th Avenue, 832 G, Miami, FL, 33136, USA. vms136@miami.edu.
CNS Drugs
|July 17, 2026
Summary
MEK inhibitors show high disease control and minimal progression for neurofibromatosis type 1 plexiform neurofibromas (NF1-PN). While tumor reduction varies, sustained disease stabilization is the primary benefit.
Area of Science:
- Oncology
- Pharmacology
- Genetics
Background:
- Plexiform neurofibromas (PN) are a major cause of morbidity in neurofibromatosis type 1 (NF1).
- MEK inhibitors are targeted therapies for NF1-PN, but their efficacy and tolerability vary.
- Previous studies show inconsistent therapeutic responses across patient populations.
Purpose of the Study:
- To systematically evaluate MEK inhibitor efficacy and safety in NF1-associated PN.
- To assess differences in treatment outcomes among key patient subgroups.
- To provide an updated analysis based on contemporary data.
Main Methods:
- A comprehensive literature search identified relevant studies on MEK inhibitors for NF1-PN.
- Random-effects meta-analysis with logit transformation was used to pool data.
- Key outcomes included objective response rate, disease control, progression, and adverse events.
Main Results:
- The study included 23 studies with 769 patients.
- Pooled objective response rate was 56%, higher in clinical trials (61%) than real-world cohorts (44%).
- Disease control rate was 96%, with only 2% progression; grade ≥3 adverse events occurred in 13% of patients.
Conclusions:
- MEK inhibitors demonstrate high disease control and minimal progression in NF1-PN.
- Therapeutic benefits include sustained disease stabilization, with response variability observed.
- Consistent effects were noted across clinical trial and real-world settings.
