Feasibility and antitumour activity of the FGFR inhibitor erdafitnib in three paediatric CNS tumour patients.
Natalia StepienLisa MayrMaria T SchmookAdalbert RaimannChristian DorferAndreas PeyrlAmedeo A AziziKathrin SchrammChristine HaberlerJohannes GojoPublished in: Pediatric blood & cancer (2024)
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.
Keyphrases
- end stage renal disease
- low grade
- ejection fraction
- newly diagnosed
- chronic kidney disease
- peritoneal dialysis
- prognostic factors
- magnetic resonance
- intensive care unit
- magnetic resonance imaging
- cell proliferation
- blood brain barrier
- patient reported outcomes
- combination therapy
- transcription factor
- brain injury
- patient reported
- white matter
- cerebral ischemia