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Molecular Profile APC T1556Nfs*3
Therapy Celecoxib + Nirogacestat
Indication/Tumor Type desmoid tumor
Response Type predicted - sensitive

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
APC T1556Nfs*3 desmoid tumor predicted - sensitive Celecoxib + Nirogacestat Case Reports/Case Series Actionable In a clinical case study, addition of Nirogacestat (PF-03084014) 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
PubMed Id Reference Title Details
(32762028) Safety and efficacy of gamma-secretase inhibitor nirogacestat (PF-03084014) in desmoid tumor: Report of four pediatric/young adult cases. Full reference...