Nirogacestat, a γ-secretase inhibitor for desmoid tumors

In a randomized, placebo-controlled trial, oral nirogacestat twice daily led to 41% of patients having a tumor response, and 2-year progression-free survival was 76%. Most adverse events were low grade.

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Bibliographic Details
Main Authors:	Gounder, Mrinal M. (Author) , Ratan, Ravin (Author) , Alcindor, Thierry (Author) , Schöffski, Patrick (Author) , Graaf, Winette T. van der (Author) , Wilky, Breelyn A. (Author) , Riedel, Richard F. (Author) , Lim, Allison (Author) , Smith, L. Mary (Author) , Moody, Stephanie (Author) , Attia, Steven (Author) , Chawla, Sant (Author) , D’Amato, Gina (Author) , Federman, Noah (Author) , Merriam, Priscilla (Author) , Tine, Brian A. Van (Author) , Vincenzi, Bruno (Author) , Benson, Charlotte (Author) , Bui, Nam Quoc (Author) , Chugh, Rashmi (Author) , Tinoco, Gabriel (Author) , Charlson, John (Author) , Dileo, Palma (Author) , Hartner, Lee (Author) , Lapeire, Lore (Author) , Mazzeo, Filomena (Author) , Palmerini, Emanuela (Author) , Reichardt, Peter (Author) , Stacchiotti, Silvia (Author) , Bailey, Howard H. (Author) , Burgess, Melissa A. (Author) , Cote, Gregory M. (Author) , Davis, Lara E. (Author) , Deshpande, Hari (Author) , Gelderblom, Hans (Author) , Grignani, Giovanni (Author) , Loggers, Elizabeth (Author) , Philip, Tony (Author) , Pressey, Joseph G. (Author) , Kummar, Shivaani (Author) , Kasper, Bernd (Author)
Format:	Article (Journal)
Language:	English
Published:	March 09, 2023
In:	The New England journal of medicine Year: 2023, Volume: 388, Issue: 10, Pages: 898-912
ISSN:	1533-4406
DOI:	10.1056/NEJMoa2210140
Online Access:	Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1056/NEJMoa2210140 Verlag, lizenzpflichtig, Volltext: https://www.nejm.org/doi/full/10.1056/NEJMoa2210140
Author Notes:	Mrinal Gounder, Ravin Ratan, Thierry Alcindor, Patrick Schöffski, Winette T. van der Graaf, Breelyn A. Wilky, Richard F. Riedel, Allison Lim, L. Mary Smith, Stephanie Moody, Steven Attia, Sant Chawla, Gina D’Amato, Noah Federman, Priscilla Merriam, Brian A. Van Tine, Bruno Vincenzi, Charlotte Benson, Nam Quoc Bui, Rashmi Chugh, Gabriel Tinoco, John Charlson, Palma Dileo, Lee Hartner, Lore Lapeire, Filomena Mazzeo, Emanuela Palmerini, Peter Reichardt, Silvia Stacchiotti, Howard H. Bailey, Melissa A. Burgess, Gregory M. Cote, Lara E. Davis, Hari Deshpande, Hans Gelderblom, Giovanni Grignani, Elizabeth Loggers, Tony Philip, Joseph G. Pressey, Shivaani Kummar, and Bernd Kasper

Description
Summary:	In a randomized, placebo-controlled trial, oral nirogacestat twice daily led to 41% of patients having a tumor response, and 2-year progression-free survival was 76%. Most adverse events were low grade.
Item Description:	Online veröffentlich: 8. März 2023 Gesehen am 15.10.2024
Physical Description:	Online Resource
ISSN:	1533-4406
DOI:	10.1056/NEJMoa2210140