TY  - JOUR
AU  - Stepien, Natalia
AU  - Mayr, Lisa
AU  - Schmook, Maria T
AU  - Raimann, Adalbert
AU  - Dorfer, Christian
AU  - Peyrl, Andreas
AU  - Azizi, Amedeo A
AU  - Schramm, Kathrin
AU  - Haberler, Christine
AU  - Gojo, Johannes
TI  - Feasibility and antitumour activity of the FGFR inhibitor erdafitnib in three paediatric CNS tumour patients.
JO  - Pediatric blood & cancer
VL  - 71
IS  - 3
SN  - 1545-5009
CY  - New York, NY
PB  - Wiley
M1  - DKFZ-2024-00057
SP  - e30836
PY  - 2024
N1  - 2024 Mar;71(3):e30836
AB  - 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.
KW  - CNS tumours (Other)
KW  - ependymoma (Other)
KW  - erdafitinib (Other)
KW  - fibroblast growth factor receptor (Other)
KW  - internal tandem duplication (ITD) (Other)
KW  - low-grade glioma (Other)
KW  - paediatrics (Other)
LB  - PUB:(DE-HGF)16
C6  - pmid:38177074
DO  - DOI:10.1002/pbc.30836
UR  - https://inrepo02.dkfz.de/record/286717
ER  -