Feasibility and antitumour activity of the FGFR inhibitor erdafitnib in three paediatric CNS tumour patients-Reference-Cited by-同舟云学术

Feasibility and antitumour activity of the FGFR inhibitor erdafitnib in three paediatric CNS tumour patients

Published:2024-01-04 Issue:3 Volume:71 Page:
ISSN:1545-5009
Container-title:Pediatric Blood & Cancer
language:en
Short-container-title:Pediatric Blood & Cancer

Author:

Stepien Natalia¹^ORCID,Mayr Lisa¹,Schmook Maria T.²,Raimann Adalbert³^ORCID,Dorfer Christian⁴,Peyrl Andreas¹^ORCID,Azizi Amedeo A.¹^ORCID,Schramm Kathrin⁵⁶,Haberler Christine⁷,Gojo Johannes¹^ORCID

Affiliation:

1. Department of Pediatrics and Adolescent Medicine Comprehensive Center for Pediatrics and Comprehensive Cancer Center Medical University of Vienna Vienna Austria

2. Division of Neuroradiology and Musculoskeletal Radiology Department of Biomedical Imaging and Image‐Guided Therapy Medical University of Vienna Vienna Austria

3. Clinical Division of Pediatric Pulmonology, Allergology and Endocrinology Department of Pediatrics and Adolescent Medicine Comprehensive Center for Pediatrics Vienna Bone and Growth Center Medical University of Vienna Vienna Austria

4. Department of Neurosurgery Medical University of Vienna Vienna Austria

5. Hopp Children's Cancer Center Heidelberg (KiTZ) Heidelberg Germany

6. Division of Pediatric Glioma Research (B360) German Cancer Research Center (DKFZ) Heidelberg Germany

7. Department of Neurology Division of Neuropathology and Neurochemistry Medical University of Vienna Vienna Austria

Abstract

AbstractAlterations 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.

Publisher

Wiley

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/pbc.30836

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