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Targeted therapy for pediatric low-grade glioma
Juan Pablo Muñoz Pérez1, Jordi Muchart2, Vicente Santa-María López1,3
1Pediatric Hematology and Oncology, Hospital Sant Joan de Déu, Barcelona, Spain.
Summary
Targeted therapy shows promise for pediatric low-grade gliomas (PLGG). Dabrafenib demonstrated higher objective response rates than trametinib, with both drugs generally well-tolerated in this study.
Area of Science:
- Pediatric Oncology
- Neuro-Oncology
- Molecular Targeted Therapy
Background:
- Pediatric low-grade gliomas (PLGG) are the most common pediatric brain tumors.
- Chemotherapy is the standard treatment for symptomatic, unresectable tumors.
- Advances in identifying molecular alterations have led to targeted drug development.
Purpose of the Study:
- To evaluate the institutional experience with MAPK pathway targeted therapy for pediatric low-grade gliomas.
- To assess the efficacy and tolerability of trametinib and dabrafenib in this patient population.
Main Methods:
- Retrospective review of 23 pediatric low-grade glioma patients treated with trametinib or dabrafenib.
- Exclusion of patients with neurofibromatosis.
- Assessment of Objective Response Rate (ORR) and Disease Control Rate (DCR) using RANO criteria.
Main Results:
- Trametinib: ORR 0%, DCR 78.6% (11 SD, 3 PD).
- Dabrafenib: ORR 41.7% (4 CR, 1 PR), DCR 100% (7 SD).
- Treatment was generally well-tolerated; Grade 3 adverse events were rare and associated with trametinib.
Conclusions:
- Targeted therapy demonstrates efficacy and tolerability in pediatric low-grade gliomas.
- Toxicity is typically mild-to-moderate and transient.
- Ongoing trials investigate the potential of targeted therapy as first-line treatment.
