TY  - JOUR
AU  - van Tilburg, Cornelis Martinus
AU  - Kilburn, Lindsay B
AU  - Perreault, Sébastien
AU  - Schmidt, Rene
AU  - Azizi, Amedeo A
AU  - Cruz-Martínez, Ofelia
AU  - Zápotocký, Michal
AU  - Scheinemann, Katrin
AU  - Meeteren, Antoinette Y N Schouten-van
AU  - Sehested, Astrid
AU  - Opocher, Enrico
AU  - Driever, Pablo Hernáiz
AU  - Avula, Shivaram
AU  - Ziegler, David S
AU  - Capper, David
AU  - Koch, Arend
AU  - Sahm, Felix
AU  - Qiu, Jiaheng
AU  - Tsao, Li-Pen
AU  - Blackman, Samuel C
AU  - Manley, Peter
AU  - Milde, Till
AU  - Witt, Ruth
AU  - Jones, David T W
AU  - Hargrave, Darren
AU  - Witt, Olaf
TI  - 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.
JO  - BMC cancer
VL  - 24
IS  - 1
SN  - 1471-2407
CY  - Heidelberg
PB  - Springer
M1  - DKFZ-2024-00242
SP  - 147
PY  - 2024
N1  - #EA:B310#LA:B310#
AB  - 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
KW  - BRAF (Other)
KW  - Chemotherapy (Other)
KW  - Child (Other)
KW  - First-line (Other)
KW  - MAPK (Other)
KW  - Pediatric low-grade glioma (Other)
KW  - Tovorafenib (Other)
KW  - pLGG (Other)
LB  - PUB:(DE-HGF)16
C6  - pmid:38291372
DO  - DOI:10.1186/s12885-024-11820-x
UR  - https://inrepo02.dkfz.de/record/287439
ER  -