Efficacy Study of Vemurafenib in Children With Recurrent or Progressive BRAFV600E- or BRAFInsT-Mutant Brain Tumors: PNOC002.

doi:10.1200/PO-26-00558

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Article

Efficacy Study of Vemurafenib in Children With Recurrent or Progressive BRAFV600E- or BRAFInsT-Mutant Brain Tumors: PNOC002.

JCO precision oncology - 1 Sept 2026

Nicolaides Theodore, Hoogendijk Raoull, Jaeger-Krause Marie, Dhall Girish, Goldman Stewart, Gajjar Amar, Kilburn Lindsay, Leary Sarah, Minturn Jane, Whipple Nicholas, Ramakrishnan Divya, Long-Boyle Janel, Aboian Mariam, Prados Michael, Molinaro Annette M, Mueller Sabine

Abstract excerpt

PURPOSE: Pediatric gliomas constitute the most prevalent central nervous system tumors in children, and aberrations in BRAF signaling contribute to their pathogenesis by disrupting the MAPK pathway. Targeted inhibition of this pathway using agents such as vemurafenib may hold therapeutic promise. PATIENTS AND METHODS: PNOC002 was a multicenter trial conducted through the Pediatric Neuro-Oncology Consortium in...

Read the complete abstract on PubMed

Topics

Adolescent
Child
Child, Preschool
Female
Humans

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Male

Young Adult

Antineoplastic Agents

Brain Neoplasms

Glioma

Mutation

Neoplasm Recurrence, Local

Proto-Oncogene Proteins B-raf

Treatment Outcome

Vemurafenib

Source

Open in PubMed Open full text

Identifiers

PMID: 42777179
DOI: 10.1200/PO-26-00558
PMCID: PMC13623076

Genes

BRAF

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 25 Oct 2026.

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