Dabrafenib plus Trametinib in Pediatric Glioma with BRAF V600 Mutations

Mené sur 110 patients pédiatriques atteints d'un gliome de faible grade présentant la mutation V600 au niveau du gène BRAF (durée médiane de suivi : 18,9 mois), cet essai de phase II évalue l'efficacité, du point de vue du taux de réponse globale, du bénéfice clinique et de la survie ans progression, et la toxicité d'un traitement de première ligne combinant dabrafénib et tramétinib

Résumé en anglais

Background: 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.

Methods: 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