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100 1 _ |a Stepien, Natalia
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245 _ _ |a Feasibility and antitumour activity of the FGFR inhibitor erdafitnib in three paediatric CNS tumour patients.
260 _ _ |a New York, NY
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500 _ _ |a 2024 Mar;71(3):e30836
520 _ _ |a 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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650 _ 7 |a erdafitinib
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650 _ 7 |a fibroblast growth factor receptor
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650 _ 7 |a internal tandem duplication (ITD)
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650 _ 7 |a low-grade glioma
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650 _ 7 |a paediatrics
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700 1 _ |a Mayr, Lisa
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700 1 _ |a Schmook, Maria T
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700 1 _ |a Raimann, Adalbert
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700 1 _ |a Dorfer, Christian
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700 1 _ |a Peyrl, Andreas
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700 1 _ |a Azizi, Amedeo A
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700 1 _ |a Schramm, Kathrin
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700 1 _ |a Haberler, Christine
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700 1 _ |a Gojo, Johannes
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773 _ _ |a 10.1002/pbc.30836
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