Disease Stabilization with MEK Inhibitors in NF1-Associated Plexiform Neurofibromas: A Systematic Review and Meta-Analysis with Subgroup Analyses by Age and Study Design.
CNS DrugsJul 17, 2026 (epub)
Sai Sanikommu, Alejandro N Santos, Bashar M Dawoud et al.
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In 30 seconds
This systematic review and meta-analysis evaluated the efficacy and safety of MEK inhibitors in 769 patients with NF1-associated plexiform neurofibromas (PN). The pooled objective response rate was 56%, with a disease control rate of 96% and a disease progression rate of 2%. The study highlights the potential of MEK inhibitors for disease stabilization in this population.
Key findings
- The pooled objective response rate for MEK inhibitors was 56% (95% CI 46-65).
- The disease control rate was 96% (95% CI 91-98).
- The disease progression rate was estimated at 2% (95% CI 1-5).
- Grade ≥ 3 adverse events occurred in 13% of patients (95% CI 6-25).
Why it matters
Understanding the efficacy and safety of MEK inhibitors in NF1-associated PN is crucial for clinicians managing this condition, as it informs treatment decisions and patient expectations regarding disease stabilization and potential adverse effects.