Reference Detail

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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Molecular Profile	Treatment Approach

Gene Name	Source	Synonyms	Protein Domains	Gene Description	Gene Role

Therapy Name	Drugs	Efficacy Evidence	Clinical Trials

Drug Name	Trade Name	Synonyms	Drug Classes	Drug Description

Gene	Variant	Impact	Protein Effect	Variant Description	Associated with drug Resistance

Molecular Profile	Indication/Tumor Type	Response Type	Therapy Name	Approval Status	Evidence Type	Efficacy Evidence	References
APC R1450* APC LOH	desmoid tumor	predicted - sensitive	Nirogacestat	Case Reports/Case Series	Actionable	In a clinical case study, Ogsiveo (nirogacestat) resulted in tumor regression in a pediatric patient with a desmoid tumor harboring APC R1450* and loss of the APC heterozygosity who stayed on treatment for 6 months (PMID: 32762028).	32762028
APC inact mut	desmoid tumor	predicted - sensitive	Nirogacestat	Case Reports/Case Series	Actionable	In a clinical case study, Ogsiveo (nirogacestat) resulted in stable disease lasting 9 months in a patient with a patient with a desmoid tumor harboring an inactivating APC mutation (PMID: 32762028).	32762028
APC T1556Nfs*3	desmoid tumor	predicted - sensitive	Celecoxib + Nirogacestat	Case Reports/Case Series	Actionable	In a clinical case study, addition of Ogsiveo (nirogacestat) to Celecoxib treatment resulted in a reduction in tumor size and stable disease that was ongoing for at least 17 months in a pediatric patient with a desmoid tumor harboring APC T1556Nfs*3 (PMID: 32762028).	32762028