Craniopharyngiomas are surgically challenging brain tumors. Postoperatively, quality of life is often significantly impaired due to neurological and endocrinological complications. Currently, FDA approved systemic treatments are not available for patients in whom craniopharyngiomas recur after surgery and radiation. Papillary craniopharyngiomas are characterized by the presence of BRAFV600E mutations. In this presentation, the authors generate an overview of all currently available information on targeted therapy in patients with BRAFV600E mutant papillary craniopharyngiomas with BRAF and/or MEK inhibitors. To date, all cases showed dramatic responses to targeted treatment with BRAF (and MEK) inhibitors. Although our data are highly informative for guiding patient treatment, uncertainty remains with regards to the optimal timing, the specific agents (single agent or dual therapy) to be used and the duration of treatment. The ongoing multicenter phase II Alliance A071601 trial (NCT03224767) of vemurafenib and cobimetinib for patients with biopsy-proven residual or recurrent papillary craniopharyngiomas should provide additional information to help inform patient management.
18 - 21 May 2019
European Society of Endocrinology