Management of selumetinib-associated toxicity in children with neurofibromatosis type 1 and plexiform neurofibromas
https://doi.org/10.17650/3034-2473-2025-2-1-30-43
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
Russian Federation
Russian Federation
Yulia Valerevna Dinikina
2 Akkuratova St., Saint Petersburg 197341
N. S. Dekhtyareva
Almazov National Medical Research Center, Ministry of Health of Russia
Russian Federation
Russian Federation
2 Akkuratova St., Saint Petersburg 197341
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Review
For citations:
Dinikina Yu.V., Dekhtyareva N.S. Management of selumetinib-associated toxicity in children with neurofibromatosis type 1 and plexiform neurofibromas. Supportive Therapy in Oncology. 2025;2(1):30-43. (In Russ.) https://doi.org/10.17650/3034-2473-2025-2-1-30-43
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