Management of selumetinib-associated toxicity in children with neurofibromatosis type 1 and plexiform neurofibromas

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Abstract

According to the statistics of major studies, neurofibromatosis is one of the most common genetic diseases associated with tumor syndrome with incidence rate of 1 case per 3000–6000 people. The most common benign neoplasms typical for neurofibromatosis type 1 are plexiform and cutaneous neurofibromas which amount to up to 95 % of all NF1-associated benign tumors.
Selumetinib is a selective mitogen-activated protein kinase types 1 and 2 inhibitor which showed promising results in the treatment of inoperable symptomatic plexiform neurofibromas. Objective response rate for selumetinib was 68 %. In 2021, selumetinib was registered in the Russian Federation and included into the State Register of Medicinal Remedies. It is important to consider that development of clinically significant adverse events during this therapy can cause drug discontinuation, reduce patients’ adherence to therapy and as a result negatively affect the overall treatment effectiveness.
The article presents a review of adverse events of selumetinib, their prevention and treatment options based on the current international guidelines.

About the authors

Yu. V. Dinikina

Almazov National Medical Research Center, Ministry of Health of Russia

Author for correspondence.
Email: dinikina_yuv@almazovcentre.ru
ORCID iD: 0000-0002-2003-0982

Yulia Valerevna Dinikina  

2 Akkuratova St., Saint Petersburg 197341 

Russian Federation

N. S. Dekhtyareva

Almazov National Medical Research Center, Ministry of Health of Russia

ORCID iD: 0000-0001-6547-0925

2 Akkuratova St., Saint Petersburg 197341 

Russian Federation

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Copyright (c) 2025 Dinikina Y.V., Dekhtyareva N.S.

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