Response to the BRAF/MEK inhibitors dabrafenib/trametinib in an adolescent with a BRAF V600E mutated anaplastic ganglioglioma intolerant to vemurafenib

Asher M Marks; Ranjit S Bindra; Michael L DiLuna; Anita Huttner; Vikram Jairam; Kristopher T Kahle; Mark W Kieran

doi:10.1002/pbc.26969

Response to the BRAF/MEK inhibitors dabrafenib/trametinib in an adolescent with a BRAF V600E mutated anaplastic ganglioglioma intolerant to vemurafenib

Pediatr Blood Cancer. 2018 May;65(5):e26969. doi: 10.1002/pbc.26969. Epub 2018 Jan 30.

Authors

Asher M Marks¹, Ranjit S Bindra², Michael L DiLuna³, Anita Huttner⁴, Vikram Jairam², Kristopher T Kahle⁵, Mark W Kieran⁶

Affiliations

¹ Department of Pediatric Hematology/Oncology, Yale University School of Medicine, New Haven, Connecticut.
² Department of Radiation Oncology, Yale University School of Medicine, New Haven, Connecticut.
³ Department of Neurosurgery, Yale University School of Medicine, New Haven, Connecticut.
⁴ Department of Pathology, Yale University School of Medicine, New Haven, Connecticut.
⁵ Department of Neurosurgery, Yale University School of Medicine.
⁶ Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.

PMID: 29380516
DOI: 10.1002/pbc.26969

Abstract

Efficacy of BRAF V600E targeted therapies in brain tumors harboring the mutation has been shown in several case reports and is currently being studied in larger clinical trials. Monotherapy with vemurafenib has been associated with significant side effects, including rashes, papillomas, and squamous cell carcinomas. Here we describe an adolescent female with anaplastic ganglioglioma and significant skin reaction to vemurafenib with subsequent tumor response and tolerance to the BRAF/MEK inhibitor combination of dabrafenib and trametinib without recurrence of previous reaction.

Keywords: AYA; BRAF V600E; BRAF inhibitor; CNS tumor; MEK inhibitor; ganglioglioma.

Publication types

Case Reports

MeSH terms

Adolescent
Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
Brain Neoplasms / drug therapy*
Brain Neoplasms / genetics
Brain Neoplasms / pathology
Drug Resistance, Neoplasm / drug effects*
Female
Ganglioglioma / drug therapy*
Ganglioglioma / genetics
Ganglioglioma / pathology
Humans
Imidazoles / administration & dosage
MAP Kinase Kinase 1 / antagonists & inhibitors*
Mutation*
Oximes / administration & dosage
Prognosis
Proto-Oncogene Proteins B-raf / antagonists & inhibitors*
Proto-Oncogene Proteins B-raf / genetics
Pyridones / administration & dosage
Pyrimidinones / administration & dosage
Vemurafenib / administration & dosage

Substances

Imidazoles
Oximes
Pyridones
Pyrimidinones
Vemurafenib
trametinib
BRAF protein, human
Proto-Oncogene Proteins B-raf
MAP Kinase Kinase 1
MAP2K1 protein, human
dabrafenib