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@ARTICLE{Stepien:286717,
author = {N. Stepien and L. Mayr and M. T. Schmook and A. Raimann and
C. Dorfer and A. Peyrl and A. A. Azizi and K. Schramm$^*$
and C. Haberler and J. Gojo},
title = {{F}easibility and antitumour activity of the {FGFR}
inhibitor erdafitnib in three paediatric {CNS} tumour
patients.},
journal = {Pediatric blood $\&$ cancer},
volume = {71},
number = {3},
issn = {1545-5009},
address = {New York, NY},
publisher = {Wiley},
reportid = {DKFZ-2024-00057},
pages = {e30836},
year = {2024},
note = {2024 Mar;71(3):e30836},
abstract = {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.},
keywords = {CNS tumours (Other) / ependymoma (Other) / erdafitinib
(Other) / fibroblast growth factor receptor (Other) /
internal tandem duplication (ITD) (Other) / low-grade glioma
(Other) / paediatrics (Other)},
cin = {B360},
ddc = {610},
cid = {I:(DE-He78)B360-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:38177074},
doi = {10.1002/pbc.30836},
url = {https://inrepo02.dkfz.de/record/286717},
}