%0 Journal Article
%A van Tilburg, Cornelis Martinus
%A Kilburn, Lindsay B
%A Perreault, Sébastien
%A Schmidt, Rene
%A Azizi, Amedeo A
%A Cruz-Martínez, Ofelia
%A Zápotocký, Michal
%A Scheinemann, Katrin
%A Meeteren, Antoinette Y N Schouten-van
%A Sehested, Astrid
%A Opocher, Enrico
%A Driever, Pablo Hernáiz
%A Avula, Shivaram
%A Ziegler, David S
%A Capper, David
%A Koch, Arend
%A Sahm, Felix
%A Qiu, Jiaheng
%A Tsao, Li-Pen
%A Blackman, Samuel C
%A Manley, Peter
%A Milde, Till
%A Witt, Ruth
%A Jones, David T W
%A Hargrave, Darren
%A Witt, Olaf
%T LOGGIC/FIREFLY-2: a phase 3, randomized trial of tovorafenib vs. chemotherapy in pediatric and young adult patients with newly diagnosed low-grade glioma harboring an activating RAF alteration.
%J BMC cancer
%V 24
%N 1
%@ 1471-2407
%C Heidelberg
%I Springer
%M DKFZ-2024-00242
%P 147
%D 2024
%Z #EA:B310#LA:B310#
%X Pediatric low-grade glioma (pLGG) is essentially a single pathway disease, with most tumors driven by genomic alterations affecting the mitogen-activated protein kinase/ERK (MAPK) pathway, predominantly KIAA1549::BRAF fusions and BRAF V600E mutations. This makes pLGG an ideal candidate for MAPK pathway-targeted treatments. The type I BRAF inhibitor, dabrafenib, in combination with the MEK inhibitor, trametinib, has been approved by the United States Food and Drug Administration for the systemic treatment of BRAF V600E-mutated pLGG. However, this combination is not approved for the treatment of patients with tumors harboring BRAF fusions as type I RAF inhibitors are ineffective in this setting and may paradoxically enhance tumor growth. The type II RAF inhibitor, tovorafenib (formerly DAY101, TAK-580, MLN2480), has shown promising activity and good tolerability in patients with BRAF-altered pLGG in the phase 2 FIREFLY-1 study, with an objective response rate (ORR) per Response Assessment in Neuro-Oncology high-grade glioma (RANO-HGG) criteria of 67
%K BRAF (Other)
%K Chemotherapy (Other)
%K Child (Other)
%K First-line (Other)
%K MAPK (Other)
%K Pediatric low-grade glioma (Other)
%K Tovorafenib (Other)
%K pLGG (Other)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ pmid:38291372
%R 10.1186/s12885-024-11820-x
%U https://inrepo02.dkfz.de/record/287439