BRAF plus MEK inhibition effective in papillary craniopharyngioma

Patients with papillary craniopharyngioma often survive several decades following diagnosis. Nonetheless, owing to the anatomical location, typically at the midline or in or above the sella turcica, patients will often present with headaches, peripheral vision loss, endocrine disorders and other neurological impairments that arise from the compression of critical structures. Surgery and/or radiotherapy can both provide disease control, albeit with a high risk of irreversible neurological damage. Now, data from a phase II trial demonstrate the feasibility of BRAF plus MEK inhibition in patients with papillary craniopharyngiomas harbouring BRAFV600E mutations, which can be detected in around 90% of these patients.

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