ESPE Yearbook of Paediatric Endocrinology

Brief Summary: This article reports a successful medical trial to test the therapeutic potential of targeting the BRAF-MEK pathway (using vemurafenib-cobimetinib combination therapy) in 16 patients with newly diagnosed papillary craniopharyngiomas (PCPs).The authors recruited 16 patients with PCP who tested positive for BRAFV600E mutation, had not undergone radiation therapy and had measurable disease. The primary endpoint was objective respons...