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Autor
Alderete, Daniel 1 Balaguer Guill, Julia 1 Baroni, Lorena 1 Bartels, Ute 1 Bavle, Abhishek 1 Bechensteen, Anne Grete 1 Bennett, Julie 1 Bornhorst, Miriam 1 Boue, Daniel R 1 Bouffet, Eric 1 Canete, Adela 1 Chintagumpala, Murali 1 Coven, Scott L 1 Cruz, Ofelia 1 Dahiya, Sonika 1 Dirks, Peter 1 Dunkel, Ira J 1 Eisenstat, David 1 Faure Conter, Cecile 1 Finch, Elizabeth 1
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Pracoviště
2nd Faculty of Medicine Charles Unive... 1 Aga Khan University Hospital Karachi ... 1 Arthur and Sonia Labatt Brain Tumour ... 1 Arthur and Sonia Labatt Brain Tumour ... 1 Children's National Health System Was... 1 Department of Hematology and Oncology... 1 Department of Hematology and Oncology... 1 Department of Hematology and Oncology... 1 Department of Hematology and Oncology... 1 Department of Laboratory Medicine and... 1 Developmental and Stem Cell Biology P... 1 Division of Pediatric Hematology and ... 1 G Gaslini Children's Hospital Genoa I... 1 Hopp Children's Cancer Center Heidelb... 1 Hospital Infantil Universitario Niño ... 1 Hospital Infantil Virgen del Rocío Se... 1 Hospital Sant Joan de Déu Barcelona S... 1 Hospital Universitario y Politecnico ... 1 Hospital of Pediatrics SAMIC Prof Dr ... 1 Institute d'Hémato Oncologie Pédiatri... 1
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Autor
Alderete, Daniel 1 Balaguer Guill, Julia 1 Baroni, Lorena 1 Bartels, Ute 1 Bavle, Abhishek 1 Bechensteen, Anne Grete 1 Bennett, Julie 1 Bornhorst, Miriam 1 Boue, Daniel R 1 Bouffet, Eric 1 Canete, Adela 1 Chintagumpala, Murali 1 Coven, Scott L 1 Cruz, Ofelia 1 Dahiya, Sonika 1 Dirks, Peter 1 Dunkel, Ira J 1 Eisenstat, David 1 Faure Conter, Cecile 1 Finch, Elizabeth 1
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Pracoviště
2nd Faculty of Medicine Charles Unive... 1 Aga Khan University Hospital Karachi ... 1 Arthur and Sonia Labatt Brain Tumour ... 1 Arthur and Sonia Labatt Brain Tumour ... 1 Children's National Health System Was... 1 Department of Hematology and Oncology... 1 Department of Hematology and Oncology... 1 Department of Hematology and Oncology... 1 Department of Hematology and Oncology... 1 Department of Laboratory Medicine and... 1 Developmental and Stem Cell Biology P... 1 Division of Pediatric Hematology and ... 1 G Gaslini Children's Hospital Genoa I... 1 Hopp Children's Cancer Center Heidelb... 1 Hospital Infantil Universitario Niño ... 1 Hospital Infantil Virgen del Rocío Se... 1 Hospital Sant Joan de Déu Barcelona S... 1 Hospital Universitario y Politecnico ... 1 Hospital of Pediatrics SAMIC Prof Dr ... 1 Institute d'Hémato Oncologie Pédiatri... 1
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PubMed
32923898
DOI
10.1200/po.19.00298
Knihovny.cz E-zdroje
PURPOSE: Children with pediatric gliomas harboring a BRAF V600E mutation have poor outcomes with current chemoradiotherapy strategies. Our aim was to study the role of targeted BRAF inhibition in these tumors. PATIENTS AND METHODS: We collected clinical, imaging, molecular, and outcome information from patients with BRAF V600E-mutated glioma treated with BRAF inhibition across 29 centers from multiple countries. RESULTS: Sixty-seven patients were treated with BRAF inhibition (pediatric low-grade gliomas [PLGGs], n = 56; pediatric high-grade gliomas [PHGGs], n = 11) for up to 5.6 years. Objective responses were observed in 80% of PLGGs, compared with 28% observed with conventional chemotherapy (P < .001). These responses were rapid (median, 4 months) and sustained in 86% of tumors up to 5 years while receiving therapy. After discontinuation of BRAF inhibition, 76.5% (13 of 17) of patients with PLGG experienced rapid progression (median, 2.3 months). However, upon rechallenge with BRAF inhibition, 90% achieved an objective response. Poor prognostic factors in conventional therapies, such as concomitant homozygous deletion of CDKN2A, were not associated with lack of response to BRAF inhibition. In contrast, only 36% of those with PHGG responded to BRAF inhibition, with all but one tumor progressing within 18 months. In PLGG, responses translated to 3-year progression-free survival of 49.6% (95% CI, 35.3% to 69.5%) versus 29.8% (95% CI, 20% to 44.4%) for BRAF inhibition versus chemotherapy, respectively (P = .02). CONCLUSION: Use of BRAF inhibition results in robust and durable responses in BRAF V600E-mutated PLGG. Prospective studies are required to determine long-term survival and functional outcomes with BRAF inhibitor therapy in childhood gliomas.
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Po ukončení testovacího provozu bude odkaz přesměrován adresu produkční verze portálu Medvik.