Feasibility and antitumour activity of the FGFR inhibitor erdafitnib in three paediatric CNS tumour patients
PEDIATRIC BLOOD & CANCER(2024)
摘要
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.
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关键词
CNS tumours,ependymoma,erdafitinib,fibroblast growth factor receptor,internal tandem duplication (ITD),low-grade glioma,paediatrics
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