TY  - JOUR
AU  - Bouffet, Eric
AU  - Hansford, Jordan R
AU  - Garrè, Maria Luisa
AU  - Hara, Junichi
AU  - Plant-Fox, Ashley
AU  - Aerts, Isabelle
AU  - Locatelli, Franco
AU  - van der Lugt, Jasper
AU  - Papusha, Ludmila
AU  - Sahm, Felix
AU  - Tabori, Uri
AU  - Cohen, Kenneth J
AU  - Packer, Roger J
AU  - Witt, Olaf
AU  - Sandalic, Larissa
AU  - Bento Pereira da Silva, Ana
AU  - Russo, Mark
AU  - Hargrave, Darren R
TI  - Dabrafenib plus Trametinib in Pediatric Glioma with BRAF V600 Mutations.
JO  - The New England journal of medicine
VL  - 389
IS  - 12
SN  - 0028-4793
CY  - Waltham, Mass.
PB  - MMS
M1  - DKFZ-2023-01911
SP  - 1108 - 1120
PY  - 2023
AB  - Detection of the BRAF V600E mutation in pediatric low-grade glioma has been associated with a lower response to standard chemotherapy. In previous trials, dabrafenib (both as monotherapy and in combination with trametinib) has shown efficacy in recurrent pediatric low-grade glioma with BRAF V600 mutations, findings that warrant further evaluation of this combination as first-line therapy.In this phase 2 trial, patients with pediatric low-grade glioma with BRAF V600 mutations who were scheduled to receive first-line therapy were randomly assigned in a 2:1 ratio to receive dabrafenib plus trametinib or standard chemotherapy (carboplatin plus vincristine). The primary outcome was the independently assessed overall response (complete or partial response) according to the Response Assessment in Neuro-Oncology criteria. Also assessed were the clinical benefit (complete or partial response or stable disease for ≥24 weeks) and progression-free survival.A total of 110 patients underwent randomization (73 to receive dabrafenib plus trametinib and 37 to receive standard chemotherapy). At a median follow-up of 18.9 months, an overall response occurred in 47
KW  - Humans
KW  - Child
KW  - Proto-Oncogene Proteins B-raf: genetics
KW  - Glioma: drug therapy
KW  - Glioma: genetics
KW  - Mutation
KW  - dabrafenib (NLM Chemicals)
KW  - trametinib (NLM Chemicals)
KW  - Proto-Oncogene Proteins B-raf (NLM Chemicals)
KW  - BRAF protein, human (NLM Chemicals)
LB  - PUB:(DE-HGF)16
C6  - pmid:37733309
DO  - DOI:10.1056/NEJMoa2303815
UR  - https://inrepo02.dkfz.de/record/283124
ER  -