Mirdametinib in symptomatic neurofibromatosis type 1 plexiform neurofibromas

Key Points

• The aim is to evaluate the effectiveness of mirdametinib for managing symptomatic neurofibromatosis type 1 plexiform neurofibromas (NF1-PNs).
• Evaluated tumor volume changes after treatment with mirdametinib
• Assessed patient-reported outcomes related to pain and quality of life
• Compared safety profiles across different age groups
• Mirdametinib led to durable reductions in tumor volume
• Significant improvements in pain and quality of life were reported
• The safety profile was manageable for various age groups

Abstract