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BRAF V600E Inhibitor (Vemurafenib) for BRAF V600E Mutated Low Grade Gliomas

Francesca Del Bufalo1, Giulia Ceglie1, Antonella Cacchione1

  • 1Department of Paediatric Haematology/Oncology, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.

Frontiers in Oncology
|November 30, 2018
PubMed

Insights

Vemurafenib shows promise in treating pediatric low-grade gliomas (LGG) with BRAF V600E mutations. This targeted therapy was generally well-tolerated and demonstrated clinical responses in most young patients with this specific genetic alteration.

Area of Science:

  • Pediatric Oncology
  • Neuro-oncology
  • Molecular Targeted Therapy

Background:

  • Low-grade gliomas (LGG) are the most frequent central nervous system tumors in children.
  • Complete surgical resection is crucial for favorable prognosis in pediatric LGG.
  • Novel therapeutic strategies are required for patients ineligible for gross total resection (GTR).

Purpose of the Study:

  • To evaluate the safety and efficacy of Vemurafenib as a single agent in pediatric patients diagnosed with BRAF V600E-mutated LGG.
  • To assess the clinical response and tolerability of Vemurafenib in this specific pediatric patient cohort.

Main Methods:

  • A cohort of 7 pediatric patients with BRAF V600E+ LGG received Vemurafenib as a single agent.
  • Treatment duration was from November 2013 to May 2018.
  • Safety, tolerability, and clinical response (including complete response, partial response, stable disease, and progression) were monitored.

Main Results:

  • Vemurafenib treatment was generally well-tolerated, with dermatological toxicity being the primary concern.
  • The study observed 1 complete response, 3 partial responses, and 1 case of stable disease among the treated patients.
  • Clinical improvement in neurological function was noted in most patients shortly after initiating therapy, though resistance mechanisms and disease progression were observed in some cases.

Conclusions:

  • Vemurafenib demonstrates potential as a treatment option for pediatric patients with BRAF V600E-mutated LGG, particularly those not amenable to GTR.
  • Despite limitations of a small cohort, the findings suggest Vemurafenib's efficacy in improving clinical outcomes for this specific group.
  • Further research is warranted to understand and overcome resistance mechanisms to optimize Vemurafenib therapy in pediatric LGG.
Keywords:
low-grade gliomaspediatric central nervous system tumorspediatric neuro-oncologytargeted therapiesvemurafenib

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