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Ref Type Journal Article
PMID (32762028)
Authors Takahashi T, Prensner JR, Robson CD, Janeway KA, Weigel BJ
Title Safety and efficacy of gamma-secretase inhibitor nirogacestat (PF-03084014) in desmoid tumor: Report of four pediatric/young adult cases.
Journal Vol Issue Date Pages Journal Short Title
Pediatric blood & cancer 67 10 2020 10 e28636 Pediatr Blood Cancer
URL
Abstract Text 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.

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