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Safety and efficacy of gamma‐secretase inhibitor nirogacestat (PF‐03084014) in desmoid tumor: Report of four pediatric/young adult cases
Author(s) -
Takahashi Takuto,
Prensner John R.,
Robson Caroline D.,
Janeway Katherine A.,
Weigel Brenda J.
Publication year - 2020
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.28636
Subject(s) - medicine , adverse effect , familial adenomatous polyposis , disease , c1 inhibitor , oncology , cancer , colorectal cancer , angioedema
Systemic therapy for pediatric desmoid tumors has been challenged by a lack of high‐quality clinical evidence and potential adverse effects. The gamma‐secretase inhibitor nirogacestat has shown promising efficacy in adults. We report four cases of pediatric and young adult desmoid tumor patients (three with familial adenomatous polyposis [FAP] syndrome) who received nirogacestat on compassionate use. After a median of 13.5 months (range 6‐18), three had durable benefit: a complete response (Case 1); a partial response (Case 2); stable disease (Case 3). The fourth had disease progression after a partial response. No patient experienced grade 3 or 4 adverse events.