The Desmoid Tumor Research Foundation

August 2020

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

Author Information

Takahashi T(1), Prensner JR(2), Robson CD(3), Janeway KA(2), Weigel BJ(1).

(1)Division of Hematology/Oncology, Dept of Pediatrics, Univ of Minnesota, Minneapolis, Minnesota. (2)Boston Children's/Dana-Farber Cancer & Blood Disorders Center, Dana-Farber Cancer Institute, Boston, Massachusetts. (3)Dept of Radiology, Boston Children's Hosp, Boston, Massachusetts.

Publication

Pediatr Blood Cancer

Summary

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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