Patient-reported pain outcomes following MEK inhibitor therapy in neurofibromatosis type 1-associated plexiform neurofibromas: A systematic review and meta-analysis.
Clin Neurol NeurosurgMay 18, 2026 (epub)
Sai Sanikommu, Alejandro N Santos, Vaidya Govindarajan et al.
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In 30 seconds
This systematic review and meta-analysis evaluated the impact of MEK inhibitors on pain outcomes in patients with neurofibromatosis type 1-associated plexiform neurofibromas. Analyzing data from 7 clinical trials with 342 patients, the study found a significant reduction in pain intensity (mean change -1.65) and pain interference (mean change -0.65), with 67% of patients reporting clinically meaningful pain reduction.
Key findings
- MEK inhibitor therapy resulted in a significant reduction in pain intensity (mean change -1.65).
- Pain interference showed a modest but significant improvement (mean change -0.65).
- 67% of patients achieved a clinically meaningful pain reduction (≥2-point decrease).
- Improvements were consistent across pediatric and adult cohorts.
Why it matters
The findings highlight the potential of MEK inhibitors to provide significant symptomatic relief for patients suffering from pain due to neurofibromatosis type 1-associated plexiform neurofibromas, which can enhance quality of life.