Safety and efficacy of gamma-secretase inhibitor nirogacestat (PF-03084014) in desmoid tumor: Report of four pediatric/young adult cases.
Pediatr Blood Cancer
; 67(10): e28636, 2020 10.
Article
em En
| MEDLINE
| ID: mdl-32762028
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.
Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Tetra-Hidronaftalenos
/
Valina
/
Fibromatose Agressiva
/
Secretases da Proteína Precursora do Amiloide
Tipo de estudo:
Prognostic_studies
Limite:
Adolescent
/
Adult
/
Child, preschool
/
Female
/
Humans
/
Male
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article