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

Jagoda Styczyńska; Agata Marjańska; Agnieszka Jatczak-Gaca; Marek Karwacki; Justyna Walenciak; Katarzyna Garus; Katarzyna Bilska; Agnieszka Mizia-Malarz; Wanda Badowska; Dorota Sławińska; Jadwiga Węcławek-Tompol; Radosław Chaber; Ewa Bień; Grażyna Karolczyk; Jolanta Skalska-Sadowska; Jan Styczyński

doi:10.5114/polp.2025.156337

eISSN: 2300-8660
ISSN: 0031-3939

Pediatria Polska - Polish Journal of Paediatrics

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abstract:

Original paper

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

Jagoda Styczyńska

¹

,

Agata Marjańska

¹

,

Agnieszka Jatczak-Gaca

¹

,

Marek Karwacki

²

,

Justyna Walenciak

³

,

Katarzyna Garus

⁴

,

Katarzyna Bilska

⁵

,

Agnieszka Mizia-Malarz

⁶

,

Wanda Badowska

⁷

,

Dorota Sławińska

⁸

,

Jadwiga Węcławek-Tompol

⁹

,

Radosław Chaber

¹⁰

,

Ewa Bień

¹¹

,

Grażyna Karolczyk

¹²

,

Jolanta Skalska-Sadowska

¹³

,

Jan Styczyński

¹

Department of Pediatric Hematology and Oncology, Collegium Medicum Nicolaus Copernicus University Toruń, Jurasz University Hospital, Bydgoszcz, Poland
Department of Pediatric Hematology and Oncology, Clinical Transplantology and Pediatrics; Medical University of Warsaw, Warszawa, Poland
Department of Pediatrics, Oncology and Hematology, Medical University of Łódź, Łódź, Poland
Department of Pediatric Oncology and Hematology, Institute of Pediatrics, Jagiellonian University Medical College, Kraków, Poland
Department of Oncology and Surgical Oncology for Children and Youth, Institute of Mother and Child, Warszawa, Poland
Department of Oncology, Hematology and Chemotherapy, Division of Pediatrics, Medical University of Silesia, Upper Silesian Child Health Centre, Katowice, Poland
Department of Pediatrics and Hematology and Oncology, Provincial Children’s Hospital, Olsztyn, Poland
Department of Pediatric Hematology and Oncology and Transplantology, Medical University of Lublin, Lublin, Poland
Department of Pediatric Hematology/Oncology and BMT, Medical University of Wrocław, Wrocław, Poland
Department of Pediatrics, Institute of Medical Sciences, Collegium Medicum, University of Rzeszów, Rzeszów, Poland
Department of Pediatrics, Hematology and Oncology, Medical University of Gdańsk, Gdańsk, Poland
Department of Pediatric Oncology and Hematology, Children’s Hospital in Kielce, Kielce, Poland
Department of Pediatric Oncology, Hematology and Transplantology, Poznań University of Medical Sciences, Poznań, Poland

Pediatr Pol 2025; 100 (4)

DOI: https://doi.org/10.5114/polp.2025.156337

Online publish date: 2025/11/20

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AMA

Styczyńska J, Marjańska A, Jatczak-Gaca A, et al. Therapy with selumetinib in children with neurofibromatosis type 1 in Poland – clinical experience of the first year of the national program. Pediatria Polska - Polish Journal of Paediatrics. 2025. doi:10.5114/polp.2025.156337.

APA

Styczyńska, J., Marjańska, A., Jatczak-Gaca, A., Karwacki, M., Walenciak, J.,  & Garus, K. et al. (2025). Therapy with selumetinib in children with neurofibromatosis type 1 in Poland – clinical experience of the first year of the national program. Pediatria Polska - Polish Journal of Paediatrics. https://doi.org/10.5114/polp.2025.156337

Chicago

Styczyńska, Jagoda, Agata Marjańska, Agnieszka Jatczak-Gaca, Marek Karwacki, Justyna Walenciak, Katarzyna Garus,  and Katarzyna Bilska et al. 2025. "Therapy with selumetinib in children with neurofibromatosis type 1 in Poland – clinical experience of the first year of the national program". Pediatria Polska - Polish Journal of Paediatrics. doi:10.5114/polp.2025.156337.

Harvard

Styczyńska, J., Marjańska, A., Jatczak-Gaca, A., Karwacki, M., Walenciak, J., Garus, K., Bilska, K., Mizia-Malarz, A., Badowska, W., Sławińska, D., Węcławek-Tompol, J., Chaber, R., Bień, E., Karolczyk, G., Skalska-Sadowska, J.,  and Styczyński, J. (2025). Therapy with selumetinib in children with neurofibromatosis type 1 in Poland – clinical experience of the first year of the national program. Pediatria Polska - Polish Journal of Paediatrics. https://doi.org/10.5114/polp.2025.156337

MLA

Styczyńska, Jagoda et al. "Therapy with selumetinib in children with neurofibromatosis type 1 in Poland – clinical experience of the first year of the national program." Pediatria Polska - Polish Journal of Paediatrics, 2025. doi:10.5114/polp.2025.156337.

Vancouver

Styczyńska J, Marjańska A, Jatczak-Gaca A, Karwacki M, Walenciak J, Garus K et al. Therapy with selumetinib in children with neurofibromatosis type 1 in Poland – clinical experience of the first year of the national program. Pediatria Polska - Polish Journal of Paediatrics. 2025. doi:10.5114/polp.2025.156337.

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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.

keywords:

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

children, neurofibromatosis type 1, plexiform neurofibroma, selumetinib