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000300325 041__ $$aEnglish
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000300325 1001_ $$aMayr, Lisa$$b0
000300325 245__ $$aEffective targeting of PDGFRA-altered high-grade glioma with avapritinib.
000300325 260__ $$aCambridge, Mass.$$bCell Press$$c2025
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000300325 520__ $$aPDGFRA is crucial to tumorigenesis and frequently genomically altered in high-grade glioma (HGG). In a comprehensive dataset of pediatric HGG (n = 261), we detect PDGFRA mutations and/or amplifications in 15% of cases, suggesting PDGFRA as a therapeutic target. We reveal that the PDGFRA/KIT inhibitor avapritinib shows (1) selectivity for PDGFRA inhibition, (2) distinct patterns of subcellular effects, (3) in vitro and in vivo activity in patient-derived HGG models, and (4) effective blood-brain barrier penetration in mice and humans. Furthermore, we report preliminary clinical real-world experience using avapritinib in pediatric and young adult patients with predominantly recurrent/refractory PDGFRA-altered HGG (n = 8). Our early data demonstrate that avapritinib is well tolerated and results in radiographic response in 3/7 cases, suggesting a potential role for avapritinib in the treatment of HGG with specific PDGFRA alterations. Overall, these translational results underscore the therapeutic potential of PDGFRA inhibition with avapritinib in HGG.
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000300325 650_7 $$2Other$$aPDGFRA alteration
000300325 650_7 $$2Other$$aPDGFRA amplification
000300325 650_7 $$2Other$$aPDGFRA inhibitor
000300325 650_7 $$2Other$$aPDGFRA mutation
000300325 650_7 $$2Other$$aavapritinib
000300325 650_7 $$2Other$$abrain penetrance
000300325 650_7 $$2Other$$adiffuse midline glioma
000300325 650_7 $$2Other$$aglioblastoma
000300325 650_7 $$2Other$$ahigh-grade glioma
000300325 650_7 $$2Other$$atyrosine kinase inhibitor
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000300325 7001_ $$aLabelle, Jenna$$b5
000300325 7001_ $$aEder, Sebastian K$$b6
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