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Verfasst von:Campos, Fernando [VerfasserIn]   i
 Kasper, Bernd [VerfasserIn]   i
Titel:Examining nirogacestat for adults with progressing desmoid tumors who require systemic treatment
Verf.angabe:Fernando Campos, and Bernd Kasper
E-Jahr:2024
Jahr:18 Oct 2024
Umfang:10 S.
Fussnoten:Gesehen am 29.04.2025
Titel Quelle:Enthalten in: Expert opinion on pharmacotherapy
Ort Quelle:Abingdon, Oxon : Routledge, Taylor & Francis, 1999
Jahr Quelle:2024
Band/Heft Quelle:25(2024), 16, Seite 2115-2124
ISSN Quelle:1744-7666
Abstract:Desmoid tumor (DT) is a rare, locally aggressive, mesenchymal neoplasm that can arise at any site in the body. Medical therapies play a major role for DT’s patients requiring treatment. A novel systemic approach has recently emerged with Nirogacestat, a γ-secretase inhibitor targeting the NOTCH signaling pathway. Nirogacestat is the first drug in its class to receive approval from the Food and Drug Administration (FDA) and is the first FDA-approved treatment specifically for DTs. We reviewed the data leading to its discovery, including its mechanism of action, pharmacological properties, clinical efficacy, and its positioning within the current treatment armamentarium for DTs. High-quality evidence for systemic therapies in the management of DTs remains an unmet need. Nirogacestat now joins sorafenib as the only drugs with efficacy in DTs demonstrated by randomized phase 3 studies. Currently, there are no comparative trials of the available systemic therapies. Therefore, physicians should consider factors such as drug accessibility, cost, toxicity profile, comorbidities, and patient preferences when selecting treatment. Long-term efficacy and safety data will be essential for evaluating the duration of treatment response and monitoring late-onset side effects of Nirogacestat.
DOI:doi:10.1080/14656566.2024.2418416
URL:Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.

Volltext: https://doi.org/10.1080/14656566.2024.2418416
 Volltext: https://www.tandfonline.com/doi/full/10.1080/14656566.2024.2418416
 DOI: https://doi.org/10.1080/14656566.2024.2418416
Datenträger:Online-Ressource
Sprache:eng
Sach-SW:aggressive fibromatosis
 desmoid fibromatosis
 Desmoid tumor
 gamma secretase inhibitor
 nirogacestat
 NOTCH pathway
K10plus-PPN:1923815636
Verknüpfungen:→ Zeitschrift

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