%0 Journal Article
%A Stepien, Natalia
%A Mayr, Lisa
%A Schmook, Maria T
%A Raimann, Adalbert
%A Dorfer, Christian
%A Peyrl, Andreas
%A Azizi, Amedeo A
%A Schramm, Kathrin
%A Haberler, Christine
%A Gojo, Johannes
%T Feasibility and antitumour activity of the FGFR inhibitor erdafitnib in three paediatric CNS tumour patients.
%J Pediatric blood & cancer
%V 71
%N 3
%@ 1545-5009
%C New York, NY
%I Wiley
%M DKFZ-2024-00057
%P e30836
%D 2024
%Z 2024 Mar;71(3):e30836
%X Alterations of the fibroblast growth factor (FGF) signalling pathway are increasingly recognized as frequent oncogenic drivers of paediatric brain tumours. We report on three patients treated with the selective FGFR1-4 inhibitor erdafitinib. Two patients were diagnosed with a posterior fossa ependymoma group A (PFA EPN) and one with a low-grade glioma (LGG), harbouring FGFR3/FGFR1 overexpression and an FGFR1 internal tandem duplication (ITD), respectively. While both EPN patients did not respond to erdafitinib treatment, the FGFR1-ITD-harbouring tumour showed a significant decrease in tumour volume and contrast enhancement throughout treatment. The tumour remained stable 6 months after treatment discontinuation.
%K CNS tumours (Other)
%K ependymoma (Other)
%K erdafitinib (Other)
%K fibroblast growth factor receptor (Other)
%K internal tandem duplication (ITD) (Other)
%K low-grade glioma (Other)
%K paediatrics (Other)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:38177074
%R 10.1002/pbc.30836
%U https://inrepo02.dkfz.de/record/286717