• Ze-Pei Wu, Yue-Long Wang, Li-Chong Wang, Zhi-Yong Liu, Rang-Rang Fan, Xin Zan, Rui-Chao Liang, Jin-Long Yang, Liang-Xue Zhou, and Jian-Guo Xu.
• Department of Neurosurgery, West China Hospital, Sichuan University, Chengdu, China.
• World Neurosurg. 2023 Dec 1; 180: e117e126e117-e126.

BackgroundAlthough a benign intracranial tumor, craniopharyngioma treatment has always been considered a challenging clinical problem. Recently, BRAF V600E mutation in the pathogenesis of papillary craniopharyngioma (PCP) has been further revealed. Thus, BRAF inhibitors (BRAFi) serve as an applicable treatment for patients with PCP.MethodsTwo patients with recurrent PCP were treated with combined BRAFi dabrafenib (150 mg, orally twice daily) and MEK inhibitors (MEKi) trametinib (2 mg, orally twice daily). A follow-up exceeding 2 years was conducted. We meticulously scrutinized the treatment's safety and efficacy profiles by delving into existing literature.ResultsOne patient harboring a solid tumor achieved a complete tumor response devoid of any adverse events and encountered no recurrence over 2 years subsequent to discontinuation. Moreover, within a mere month of commencing targeted therapy, the tumor demonstrated observable shrinkage. This finding substantiates the considerable potential inherent in targeted therapy for PCP cases marked by the somatic BRAF V600E mutation.ConclusionsUnder specific conditions, individuals diagnosed with PCP can attain a complete tumor response following combined treatment with BRAFi/MEKi.Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.

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