Peer-influenced content. Sources you trust. No registration required. This is HCN.

The New England Journal of MedicineDabrafenib plus Trametinib in Pediatric Glioma with BRAF V600 Mutations

The combination therapy shines in a Phase 2 trial

In a quest to optimize treatment for pediatric low-grade glioma patients with BRAF V600 mutations, a phase 2 clinical trial offers compelling evidence that dabrafenib in combination with trametinib outperforms standard chemotherapy in terms of response rate, progression-free survival, and safety profile.

HCN Medical Memo
This study underscores the potential of using dabrafenib plus trametinib as an effective and safer first-line therapy compared to standard chemotherapy. This combination therapy demonstrates a notably higher response rate and extended progression-free survival, positioning it as a new standard of care that could reshape clinical decision-making.

Study Design
  • Phase 2 clinical trial involving pediatric patients with low-grade glioma and BRAF V600 mutations.
  • 110 patients were randomly assigned in a 2:1 ratio: 73 to receive dabrafenib plus trametinib, and 37 to receive standard chemotherapy (carboplatin plus vincristine).
  • Primary outcome measured was the independently assessed overall response, along with clinical benefit and progression-free survival.

Pediatric low-grade gliomas constitute up to 30% of all childhood central nervous system tumors.

Key Findings
  • Overall response in the dabrafenib plus trametinib group was 47%, compared to 11% in the standard chemotherapy group (Risk ratio, 4.31; P<0.001).
  • Clinical benefit was noted in 86% of the patients on dabrafenib plus trametinib vs. 46% on standard chemotherapy (Risk ratio, 1.88).
  • Median progression-free survival was significantly longer in the combination therapy group: 20.1 months vs. 7.4 months (Hazard ratio, 0.31; P<0.001).
  • Grade 3 or higher adverse events were noted in 47% of the patients on combination therapy and 94% on chemotherapy.

More in Pediatric Cancer

The Healthcare Communications Network is owned and operated by IQVIA Inc.

Click below to leave this site and continue to IQVIA’s Privacy Choices form