Safety and efficacy of gamma-secretase inhibitor nirogacestat (PF-03084014) in desmoid tumor: Report of four pediatric/young adult cases

Takuto Takahashi, John R Prensner, Caroline D Robson, Katherine A Janeway, Brenda J Weigel

Research output: Contribution to journalArticlepeer-review

2 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)e28636
JournalPediatric blood & cancer
DOIs
StatePublished - Aug 6 2020

Bibliographical note

© 2020 Wiley Periodicals LLC.

PubMed: MeSH publication types

  • Journal Article
  • Case Reports

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