Priscilla K Brastianos, Erin Twohy, Susan Geyer, Elizabeth R Gerstner, Timothy J Kaufmann, Shervin Tabrizi, Brian Kabat, Julia Thierauf, Michael W Ruff, Daniela A Bota, David A Reardon, Adam L Cohen, Macarena I De La Fuente, Glenn J Lesser, Jian Campian, Pankaj K Agarwalla, Priya Kumthekar, Bhupinder Mann, Shivangi Vora, Michael Knopp, A John Iafrate, William T Curry, Daniel P Cahill, Helen A Shih, Paul D Brown, Sandro Santagata, Fred G Barker, Evanthia Galanis
BACKGROUND: Craniopharyngiomas, primary brain tumors of the pituitary-hypothalamic axis, can cause clinically significant sequelae. Treatment with the use of surgery, radiation, or both is often associated with substantial morbidity related to vision loss, neuroendocrine dysfunction, and memory loss. Genotyping has shown that more than 90% of papillary craniopharyngiomas carry BRAF V600E mutations, but data are lacking with regard to the safety and efficacy of BRAF-MEK inhibition in patients with papillary craniopharyngiomas who have not undergone previous radiation therapy...
July 13, 2023: New England Journal of Medicine