Adjuvant Treatment...Useful in Certain Circumstance...Pilocytic astrocytoma, PXA, ganglioglioma if BRAF V600E activating mutation…vemurafenib…

ZELBORAF is a kinase inhibitor indicated for the treatment of patients with unresectable or metastatic melanoma with BRAF V600E mutation as detected by an FDA-approved test.

We present three pediatric patients with BRAFV600E mutant high-grade gliomas treated by vemurafenib on a nominative authorization level at our institution. One patient with anaplastic ganglioglioma experienced confirmed partial tumor response and significant clinical improvement and she is alive 20 months after start of treatment. 

We report on a case of BRAFV600E mutated cervicomedullary GG treated with standard chemotherapy and surgery. After multiple relapse, BRAF status was analyzed by immunohistochemistry and sequencing showing a BRAFV600E mutation. Treatment with Vemurafenib as single agent was started. For the first time, a radiological and clinical response was obtained after 3 months of treatment and sustained after 6 months.

We describe a case of an adult with a progressive BRAF V600E mutant spinal cord ganglioglioma 9 years after surgery who was treated with vemurafenib....After 8 weeks of treatment, the patient was neurologically stable and brain MRI showed a >50% decrease in tumor size (Figure 1). A steady partial response was observed for more than 13 months....Twenty-one months after vemurafenib discontinuation in October 2018, MRI and neurological examination showed stable disease (Figure 1) and the patient had no side effects.

A 13-year-old girl presented to our clinic in March 2010 with complaints of progressive loss of tactile sensation in the right upper and lower extremities….A partial resection was obtained and pathology was consistent with a ganglioglioma....Review of the molecular characteristics of the original tumor demonstrated the presence of a BRAF V600E mutation....she was started on vemurafenib 960 mg orally twice per day along with vinblastine 2 mg/m2 once per week....Twelve weeks after initiation of therapy, an MRI scan (Fig 1B) revealed a continued overall decrease in the size of the lesion,...