# Dabrafenib plus trametinib in relapsed or refractory BRAF V600-mutant paediatric high-grade glioma

Source: https://onco.cc/key-papers/paper-hargrave-dabrafenib-trametinib-paediatric-hgg-jco-2023/  
OnCo record `paper-hargrave-dabrafenib-trametinib-paediatric-hgg-jco-2023` (Key paper). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

In children whose BRAF V600-mutant high-grade glioma had relapsed, the combination of dabrafenib and trametinib shrank tumours in more than half and gave responses lasting well over a year, far better than historical chemotherapy.

## Summary

Phase 2 study of 41 children and adolescents with relapsed or refractory BRAF V600-mutant high-grade glioma treated with dabrafenib plus trametinib.

Objective response by independent review was 56 percent with a median duration of response of 22.2 months and median progression-free survival of 9.0 months, compared with expected responses of under 20 percent with chemotherapy; toxicity was mostly fever, rash and gastrointestinal.

## Fields

- Kind: Key paper
- Last checked: 2026-09-17
- Journal: Journal of Clinical Oncology
- Year: 2023
- DOI: 10.1200/JCO.23.00558
- Authors: Hargrave DR, Terashima K, Hara J, et al.
- Findings: Objective response 56 percent; median duration of response 22.2 months.; Median overall survival 32.8 months.
- What it means: BRAF V600E testing is mandatory in childhood high-grade glioma and the combination is a standard at relapse and, increasingly, alongside or before radiotherapy in newly diagnosed disease; it supported the tumour-agnostic approval in children.
- Caveats: Small single-arm cohort.; Resistance eventually develops in most patients.

## Sources

- J Clin Oncol 2023: https://doi.org/10.1200/JCO.23.00558
- PubMed: https://pubmed.ncbi.nlm.nih.gov/37643378/

## Connected records

- cancers: [Paediatric high-grade glioma (excluding diffuse midline glioma)](https://onco.cc/cancers/paediatric-high-grade-glioma/)
- drugs: [Dabrafenib](https://onco.cc/drugs/dabrafenib/), [Trametinib](https://onco.cc/drugs/trametinib/)
- journals: [Journal of Clinical Oncology](https://onco.cc/journals/jco/)

---
JSON: https://onco.cc/api/v1/entities/paper-hargrave-dabrafenib-trametinib-paediatric-hgg-jco-2023.json