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

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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.

PlumX metrics:

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2. De Blank PMK, Gross AM, Akshintala S, Blakeley Jo, Bollag G, Cannon A, et al. MEK inhibitors for neurofibromatosis type 1 manifestations: clinical evidence and consensus. Neuro Oncol 2022; 24: 1845-1856.

3. Kim H, Yoon HM, Kim EK, Ra YS, Kim HW, Yum MS, et al. Safety and efficacy of selumetinib in pediatric and adult patients with neurofibromatosis type 1 and plexiform neurofibroma. Neuro Oncol 2024; 26: 2352-2363.

4. Armstrong AE, Belzberg AJ, Crawford JR, Hirbe AC, Wang ZJ. Treatment decisions and the use of MEK inhibitors for children with neurofibromatosis type 1-related plexiform neurofibromas. BMC Cancer 2023; 23: 553.

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7. Azizi AA, Hargrave D, Passos J, Wolkenstein P, Rosenbaum T, Santoro C, et al. Consensus recommendations on management of selumetinib-associated adverse events in pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas. Neurooncol Pract 2024; 11: 515-531.

8. Płużański A. Evaluation of response to treatment – criteria RECIST 1.1. J Oncol 2014; 64: 331-335..

9. NIH NCI DCTD Cancer Evaluation Program. Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. Available from: https://ctep.cancer.gov/protocoldevelopment/electronic_applications/docs/ctcae_v5_quick_ reference_8.5x11.pdf (accessed: 20.05.2025).

10. Gross AM, Wolters PL, Dombi E, Baldwin A, Whitcomb P, Fisher MJ, et al. Selumetinib in children with inoperable plexiform neurofibromas. N Engl J Med 2020; 382: 1430-1442.

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