Journal Article

DKFZ-2024-00057

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Feasibility and antitumour activity of the FGFR inhibitor erdafitnib in three paediatric CNS tumour patients.

Stepien, N. ; Mayr, L. ; Schmook, M. T. ; Raimann, A. ; Dorfer, C. ; Peyrl, A. ; Azizi, A. A. ; Schramm, K.DKFZ* ; Haberler, C. ; Gojo, J.

2024
Wiley New York, NY

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Please use a persistent id in citations: doi:10.1002/pbc.30836

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.

Keyword(s): CNS tumours ; ependymoma ; erdafitinib ; fibroblast growth factor receptor ; internal tandem duplication (ITD) ; low-grade glioma ; paediatrics

Note: 2024 Mar;71(3):e30836

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Contributing Institute(s):

1. Pädiatische Gliomforschung (B360)

Research Program(s):

1. 312 - Funktionelle und strukturelle Genomforschung (POF4-312) (POF4-312)

Appears in the scientific report 2024

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Database coverage:
; BIOSIS Previews ; Biological Abstracts ; Clarivate Analytics Master Journal List ; Current Contents - Clinical Medicine ; DEAL Wiley ; Essential Science Indicators ; IF < 5 ; JCR ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

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