perinatologyРоссийский вестник перинатологии и педиатрииRossiyskiy Vestnik Perinatologii i Pediatrii (Russian Bulletin of Perinatology and Pediatrics)1027-40652500-2228Ltd. “The National Academy of Pediatric Science and Innovation”10.21508/1027-4065-2025-70-5-5-13perinatology-2263Research ArticleОБЗОРЫ ЛИТЕРАТУРЫLITERATURE REVIEWSТаргетная терапия неоперабельных плексиформных нейрофибром у детей с нейрофиброматозом 1-го типаTargeted therapy for inoperable plexiform neurofibromas in children with neurofibromatosis type 1https://orcid.org/0000-0002-4147-2309ВолгинаС. Я.VolginaS. Ya.

Волгина Светлана Яковлевна — д.м.н., проф. кафедры госпитальной педиатрии.

420012, Казань, ул. Бутлерова, д.49

420012, Kazan

volgina_svetlana@mail.ruhttps://orcid.org/0009-0005-8926-0136КурбановаЕ. С.KurbanovaYa. S.

Курбанова Есуман Сайбилоловна — ординатор кафедры госпитальной педиатрии.

420012, Казань, ул. Бутлерова, д.49

420012, Kazan

https://orcid.org/0000-0002-8582-592XГамироваР. Г.GamirovaR. G.

Гамирова Римма Габдульбаровна — к.м.н., доцент, зав. кафедрой неврологии с курсами психиатрии, клинической психологии и медицинской генетики, ведущий научный сотрудник НИЛ «Нейрокогнитивные исследования».

420008, Казань, ул. Кремлевская, д.18

420008, Kazan

https://orcid.org/0000-0002-9687-4583СоловьеваН. A.SolovyevaN. A.

Соловьева Наиля Анасовна — к.м.н., доцент кафедры госпитальной педиатрии.

420012, Казань, ул. Бутлерова, д.49

420012, Kazan

https://orcid.org/0000-0003-1741-2629КулаковаГ. А.KulakovaG. A.

Кулакова Галина Александровна — к.м.н., доцент кафедры госпитальной педиатрии.

420012, Казань, ул. Бутлерова, д.49

420012, Kazan

https://orcid.org/0000-0003-0873-8037КурмаеваЕ. А.KurmayevaE. A.

Курмаева Елена Анатольевна — к.м.н., доцент кафедры госпитальной педиатрии.

420012, Казань, ул. Бутлерова, д.49

420012, Kazan

ФГБОУ ВО «Казанский государственный медицинский университет» Минздрава РоссииРоссияKazan State Medical UniversityRussian FederationФГАОУ ВО Казанский (Приволжский) федеральный университетРоссияKazan (Volga region) Federal UniversityRussian Federation202514112025705513Copyright © Ltd. “The National Academy of Pediatric Science and Innovation”, 20252025Ltd. “The National Academy of Pediatric Science and Innovation”Ltd. “The National Academy of Pediatric Science and Innovation”https://www.ped-perinatology.ru/jour/about/submissions#copyrightNoticehttps://www.ped-perinatology.ru/jour/article/view/2263

Нейрофиброматоз 1 типа является мультисистемным генетическим заболеванием, характеризующимся развитием доброкачественных и злокачественных опухолей, среди которых особое место занимают плексиформные нейрофибромы. Эти опухоли, возникающие у 10–30% детей с нейрофиброматозом 1 типа, существенно ухудшают качество жизни, вызывая боль, обезображивание и функциональные нарушения. Традиционное лечение плексиформных нейрофибром ограничивалось хирургическим вмешательством, однако возможность радикальной резекции опухолей существует лишь у небольшого числа пациентов. Применение селективного ингибитора MEK — селуметиниба открыло новый этап терапии неоперабельных плексиформных нейрофибром. Клинические исследования демонстрируют значительное уменьшение объема опухоли (у 68 % пациентов наблюдался частичный ответ с длительным улучшением) и улучшение клинических симптомов, таких как боль и двигательные нарушения. Несмотря на преимущественно легкую или умеренную токсичность лекарственного препарата, наиболее распространенные нежелательные явления включали кожные реакции, желудочно-кишечные расстройства и повышение уровня креатинфосфокиназы, требующие тщательного мониторинга и своевременной коррекции. Введение селуметиниба в клиническую практику значительно изменило подход к ведению пациентов с нейрофиброматозом 1 типа, позволяя эффективно контролировать рост плексиформных нейрофибром, улучшать качество жизни пациентов и минимизировать необходимость повторных травматичных хирургических вмешательств. Тем не менее, требуется дальнейшее изучение оптимальных доз и длительности терапии.

Neurofibromatosis type 1 is a multisystem genetic disorder characterized by the development of benign and malignant tumors, among which plexiform neurofibromas occupy a special place. These tumors, which occur in 10–30% of patients, significantly impair the quality of life, causing pain, disfigurement and functional impairment. Traditional treatment of plexiform neurofibromas was limited to surgical intervention, but the possibility of radical resection of tumors exists only in a small number of patients. The use of the selective MEK inhibitor selumetinib has opened a new stage of therapy for inoperable plexiform neurofibromas. Clinical studies demonstrate a significant reduction in tumor volume (68% of patients showed a partial response with long-term improvement) and improvement in clinical symptoms, such as pain and motor impairment. Despite predominantly mild or moderate toxicity, the most common adverse events included skin reactions, gastrointestinal disorders, and increased creatine phosphokinase levels, requiring careful monitoring and timely correction. The introduction of selumetinib into clinical practice has significantly changed the approach to managing patients with neurofibromatosis type 1, allowing for effective control of plexiform neurofibromas growth, improving the quality of life of patients, and minimizing the need for repeated traumatic surgical interventions. However, further study of optimal doses, duration of treatment, and monitoring of adverse events during targeted therapy of inoperable plexiform neurofibromas in children with neurofibromatosis type 1 is required.

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The authors declare that there are no conflicts of interest present.