ArticleNeuro-oncology2022
Upfront molecular targeted therapy for the treatment of BRAF-mutant pediatric high-grade glioma.
Article in Neuro-oncology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers, 4 of them syntheses that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
40 citing papers in PubMed, 4 syntheses or guidelines pooled it, 59 citations in OpenAlex.
- Efficacy and safety of dabrafenib plus trametinib in pediatric versus adult gliomas: a systematic review and meta-analysis.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2025Pooled it
- Dabrafenib plus trametinib in low-grade versus high-grade gliomas: a systematic review and meta-analysis.BMC cancer · 2024Pooled it
- The effects of dabrafenib and/or trametinib treatment in Braf V600-mutant glioma: a systematic review and meta-analysis.Neurosurgical review · 2024Pooled it
- Systematic Review of Molecular Targeted Therapies for Adult-Type Diffuse Glioma: An Analysis of Clinical and Laboratory Studies.International journal of molecular sciences · 2023Pooled it
- Trial
- Phase II Trial of Dabrafenib Plus Trametinib in Relapsed/RefractoryJournal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023Trial
- Practical management of BRAF inhibitors in glioma: toxicity and resistance.CNS oncology · 2026Review
- The future of targeted therapies for pediatric central nervous system tumors.Neuro-oncology advances · 2026Review
- RAS/MAPK inhibition in pediatric gliomas.Neuro-oncology advances · 2026Review
- Targeted therapies in adolescent and young adult patients with central nervous system tumors.Neuro-oncology advances · 2026Review
- A bibliometric analysis of progress and trends in pediatric glioma research.Discover oncology · 2026Article
- From Molecular Alterations to the Targeted Therapy: Treatment of Thalamic Glioma in Pediatric Patients.International journal of molecular sciences · 2026Review
- Family and Physician Perspectives: Unassailable, a p53 and PIK3CA Mutant Glioblastoma in a Child.Current oncology (Toronto, Ont.) · 2025Article
- The great imitator: clinical, surgical, and molecular insights into diffuse midline gliomas, H3 K27-altered. Illustrative cases.Journal of neurosurgery. Case lessons · 2025Article
- Dabrafenib in pediatric patients withNeuro-oncology practice · 2025Article
- Point/counterpoint: Upfront BRAF inhibition for adult BRAF-mutant high-grade gliomas.Neuro-oncology · 2025Article
- Targeted Inhibition in Pediatric MET and ALK-Altered Hemispheric Gliomas: Objective Responses Followed by Treatment Resistance.International journal of molecular sciences · 2025Article
- Comparative analysis of RNA expression identifies effective targeted drug in myoepithelial carcinoma.NPJ precision oncology · 2025Article
- Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule Inhibitors.Current oncology (Toronto, Ont.) · 2025Review
- Targeted therapy for pediatric glioma: RAF(t)ing in the molecular era.World journal of pediatrics : WJP · 2025Review
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Authors and funding
25 authors at 11 institutions in 1 country.
Funding
Abstract
backgroundThe prognosis for patients with pediatric high-grade glioma (pHGG) is poor despite aggressive multimodal therapy. Objective responses to targeted therapy with BRAF inhibitors have been reported in some patients with recurrent BRAF-mutant pHGG but are rarely sustained.
methodsWe performed a retrospective, multi-institutional review of patients with BRAF-mutant pHGG treated with off-label BRAF +/- MEK inhibitors as part of their initial therapy.
resultsNineteen patients were identified, with a median age of 11.7 years (range, 2.3-21.4). Histologic diagnoses included HGG (n = 6), glioblastoma (n = 3), anaplastic ganglioglioma (n = 4), diffuse midline glioma (n = 3), high-grade neuroepithelial tumor (n = 1), anaplastic astrocytoma (n = 1), and anaplastic astroblastoma (n = 1). Recurrent concomitant oncogenic alterations included CDKN2A/B loss, H3 K27M, as well as mutations in ATRX, EGFR, and TERT. Eight patients received BRAF inhibitor monotherapy. Eleven patients received combination therapy with BRAF and MEK inhibitors. Most patients tolerated long-term treatment well with no grade 4-5 toxicities. Objective and durable imaging responses were seen in the majority of patients with measurable disease. At a median follow-up of 2.3 years (range, 0.3-6.5), three-year progression-free and overall survival for the cohort were 65% and 82%, respectively, and superior to a historical control cohort of BRAF-mutant pHGG patients treated with conventional therapies.
conclusionsUpfront targeted therapy for patients with BRAF-mutant pHGG is feasible and effective, with superior clinical outcomes compared to historical data. This promising treatment paradigm is currently being evaluated prospectively in the Children's Oncology Group ACNS1723 clinical trial.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.