Case Report: Nirogacestat therapy induces rapid response in a patient with refractory, life-threatening desmoid tumor

URN to cite this document:: urn:nbn:de:bvb:355-epub-782436
DOI to cite this document:: 10.5283/epub.78243

Harrer, Dennis Christoph

; Herrmann, Markus ; Vogelhuber, Martin ; Geis, Sebastian ; Mahr, Daniel ; Niethard, Maya ; Keil, Felix

; Napodano, Gerardo ; Herr, Wolfgang ; Grube, Matthias

License: Creative Commons Attribution 4.0
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Date of publication of this fulltext: 28 Nov 2025 16:29

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Abstract

Background: Desmoid tumor (DT) is a rare soft tissue neoplasm characterized by locally aggressive infiltration into adjacent tissues with few established treatment regimens. So far, the evidence-based therapeutic arsenal for DT has comprised surgery, locoregional therapy, tyrosine kinase inhibitor treatment, and chemotherapy. The γ-secretase inhibitor nirogacestat was approved by the FDA in 2023 ...