Background: To explore the effect of BRAF inhibitor on epithelioid glioblastoma (Ep-GBM) with BRAFV600E mutation.
Methods: A patient of Ep-GBM with BRAFV600E mutation underwent BRAF inhibition therapy. The rationale behind combined BRAF and MEK inhibition in Ep-GBM was reviewed.
Results: Vemurafenib can initially inhibit the progression of Ep-GBM with BRAFV600E mutation. However, the tumor may become resistant to vemurafenib and then progress.
Conclusions: BRAF inhibition therapy can inhibit the progression of Ep-GBM with BRAFV600E mutation, but the subsequent resistance development leads to a poor outcome.