As these targetable mutations warrant prospective research, the authors will be conducting a national National Cancer Institute-sponsored multicenter clinical trial to investigate BRAF/MEK inhibition in the treatment of craniopharyngioma.
In total, 14-50% of adult-onset craniopharyngioma are papillary; the majority with a mutation in exon 3 of BRAF and may respond to BRAF inhibitors and mitogen-activated protein kinase inhibitors.
Here, we describe our treatment of a man age 39 years with multiply recurrent BRAFV600Ecraniopharyngioma using dabrafenib (150mg, orally twice daily) and trametinib (2mg, orally twice daily).