Therapy with selumetinib in children with neurofibromatosis type 1 in Poland – clinical experience of the first year of the national program

Introduction Plexiform neurofibroma (PN) is one of the predominant symptoms of neurofibromatosis type 1 (NF1), being responsible for significant morbidity. Medical care of children with NF1 in Poland is supervised by pediatric oncologists. Since 2020 the coordinating medical care (CKOM) pilot program has been held in Poland, consisting of/covering 6 pediatric oncology centers (Bydgoszcz, Gdańsk, Olsztyn, Kraków and 2 centers in Warszawa). Since 2024 the treatment for NF1-related PNs with selumetinib (MEK inhibitor) in pediatric patients has been reimbursed due to the national therapeutic program (B.155) provided by the National Health Fund and offered in up to now in 15 pediatric oncology departments, including CKOMs. The aim of this study was to analyze the effectiveness of the national therapeutic program in the first fourteen months of its activity. Material and methods A total of 79 pediatric patients were included in the national program from January 2024 to the end of February 2025. We analyzed demographics, tumor location and size. The preliminary data also included adverse effects and the effectiveness of therapy. Patients were assessed before the inclusion into the program and periodically in 4-month periods of treatment. Results Overall, partial regression was observed in 35.3% patients already after 8 months of therapy with selumetinib, including 17.6% of them who met the criteria of confirmed partial response; one patient experienced progression of the lesion. The most frequently reported adverse effects included the acne-like rash; only one patient required permanent dose reduction of selumetinib. Conclusions The findings of our study align with those of the SPRINT study; no additional safety concerns emerged.