Comparison of Early Response to Selumetinib Therapy in Adolescents and Children With NF1-related Plexiform Neurofibromas.

BACKGROUND/AIM: Following approval from the European Medicines Agency, selumetinib has emerged as a targeted therapy for pediatric patients aged ≥3 years with inoperable, symptomatic NF1-related plexiform neurofibromas (PNs). In Poland, this treatment has been reimbursed since January 2024 under a national therapeutic program funded by the National Health Service. Nevertheless, the efficacy and safety of selumetinib in subgroups within the pediatric population remain unclear. This study aimed to evaluate the efficacy and safety of selumetinib treatment in adolescents (≥16 years old) compared with younger children with NF1-related PNs. PATIENTS AND METHODS: A total of 111 patients were included in the program over a two-year period. Patients were divided into two groups according to age: adolescents (≥16 years of age) and children (<16 years of age). Demographic characteristics, PN location, treatment efficacy and adverse effects were analyzed and compared between the two groups. RESULTS: =0.035). Skin-related adverse effects were the most common in both groups, and only isolated cases required selumetinib dose reduction. CONCLUSION: Early response to selumetinib therapy is achieved faster in children than in adolescents. Selumetinib is generally well tolerated in both age groups.