Efficacy and safety of MEK inhibitors for NF1-associated symptomatic, inoperable plexiform neurofibromas: A systematic review and meta-analysis.

In 30 seconds

This systematic review and meta-analysis evaluated the efficacy and safety of MEK inhibitors for treating symptomatic, inoperable plexiform neurofibromas in patients with neurofibromatosis type 1 (NF1). Analyzing data from 528 participants across nine studies, the pooled objective response rate was 53.7%, with 62.9% in children and 40.8% in adults. The study highlights the potential of MEK inhibitors in managing this condition.

Key findings

• The pooled objective response rate for MEK inhibitors was 53.7% (95% CI 40.5-66.6).
• Response rates varied by age: 62.9% in children versus 40.8% in adults.
• The only randomized trial showed a response rate of 20% for MEK inhibitors compared to 5% for placebo (p = 0.011).
• Toxicity was mostly low grade, with rare occurrences of cardiac and ocular events.

Why it matters

The findings support the use of MEK inhibitors as a treatment option for symptomatic, inoperable plexiform neurofibromas in NF1 patients, providing a potential avenue for improving patient quality of life and managing symptoms.