Surgery and OncologySurgery and OncologyХирургия и онкология2949-5857Publishing House ABV Press84110.17650/2949-5857-2026-16-2-44-53REVIEWОБЗОРЫ ЛИТЕРАТУРЫReview ArticleNirogacestat in treatment of patients with desmoid tumors. Literature reviewНирогацестат в лечении больных с десмоидными опухолями. Обзор литературыhttps://orcid.org/0000-0001-8887-5108TurupaevK. A.ТурупаевК. А.
Russian Federation
Kir-turupaev@yandex.ruhttps://orcid.org/0009-0007-1861-3306GrebennikovaO. P.ГребенниковаО. П.
Russian Federation
Kir-turupaev@yandex.ruhttps://orcid.org/0000-0003-1391-0516BudurovaM. D.БудуроваМ. Д.
Russian Federation
Kir-turupaev@yandex.ruhttps://orcid.org/0009-0009-5878-6951LadykoD. D.ЛадыкоД. Д.
Russian Federation
Kir-turupaev@yandex.ruSelchukV. Y.СельчукВ. Ю.
Russian Federation
Kir-turupaev@yandex.ruN.N. Blokhin National Medical Research Center of Oncology, Ministry of Health of RussiaФГБУ «Национальный медицинский исследовательский центр онкологии им. Н. Н. Блохина» Минздрава РоссииRussian University of Medicine, Ministry of Health of RussiaФГБОУ ВО «Российский университет медицины» Минздрава РоссииP.A. Hertzen Moscow Oncology Research Institute – branch of the National Medical Research Radiological Center, Ministry of Health of RussiaМосковский научно-исследовательский онкологический институт им. П. А. Герцена – филиал ФГБУ «Национальный медицинский исследовательский центр радиологии» Минздрава России20062026162445309092025Copyright ©; 2026, ABV-pressCopyright ©; 2026, АБВ-пресс2026ABV-pressАБВ-прессhttps://onco-surgery.info/jour/about/editorialPolicieshttps://onco-surgery.info/jour/article/view/841

This literature review analyzes the role of the gamma-secretase inhibitor nirogacestat in treating patients with progressive desmoid tumors. The article systematizes data on the molecular mechanisms of the drug's action, stages of its clinical development, and the final results of the phase III DeFi trial, which formed the basis for nirogacestat's regulatory approval. It is shown that the drug significantly improves progression-free survival, increases objective response rate, and leads to clinically meaningful improvement in quality of life. The review discusses nirogacestat's safety profile in detail, highlighting a specific yet reversible adverse event – ovarian dysfunction. The analyzed data provide evidence for the efficacy and safety of nirogacestat, supporting its consideration as a new therapeutic option for patients with progressive desmoid fibromatosis.

Настоящий обзор литературы посвящен анализу роли ингибитора γ-секретазы нирогацестата в лечении пациентов с прогрессирующими десмоидными опухолями. В работе систематизированы данные о молекулярных механизмах действия препарата, этапах его клинического развития и финальных результатах исследования III фазы DeFi, которые легли в основу одобрения нирогацестата регулирующими органами. Показано, что препарат достоверно улучшает выживаемость без прогрессирования, увеличивает частоту объективных ответов и приводит к клинически значимому улучшению качества жизни. В обзоре детально обсуждается профиль безопасности нирогацестата, акцентируется внимание на специфическом, но обратимом нежелательном явлении – дисфункции яичников. Обзор свидетельствует об эффективности и безопасности нирогацестата, что позволяет рассматривать его в качестве новой опции терапии больных прогрессирующим десмоидным фиброматозом.

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