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000286717 1001_ $$00000-0002-6658-8367$$aStepien, Natalia$$b0
000286717 245__ $$aFeasibility and antitumour activity of the FGFR inhibitor erdafitnib in three paediatric CNS tumour patients.
000286717 260__ $$aNew York, NY$$bWiley$$c2024
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000286717 520__ $$aAlterations 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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000286717 650_7 $$2Other$$aCNS tumours
000286717 650_7 $$2Other$$aependymoma
000286717 650_7 $$2Other$$aerdafitinib
000286717 650_7 $$2Other$$afibroblast growth factor receptor
000286717 650_7 $$2Other$$ainternal tandem duplication (ITD)
000286717 650_7 $$2Other$$alow-grade glioma
000286717 650_7 $$2Other$$apaediatrics
000286717 7001_ $$aMayr, Lisa$$b1
000286717 7001_ $$aSchmook, Maria T$$b2
000286717 7001_ $$00000-0002-3551-594X$$aRaimann, Adalbert$$b3
000286717 7001_ $$aDorfer, Christian$$b4
000286717 7001_ $$00000-0002-5736-8231$$aPeyrl, Andreas$$b5
000286717 7001_ $$00000-0002-1347-6644$$aAzizi, Amedeo A$$b6
000286717 7001_ $$0P:(DE-He78)2f592d9d8339bee07cca3956b7472b61$$aSchramm, Kathrin$$b7$$udkfz
000286717 7001_ $$aHaberler, Christine$$b8
000286717 7001_ $$aGojo, Johannes$$b9
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