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Ref Type Journal Article
PMID (37683921)
Authors Wu ZP, Wang YL, Wang LC, Liu ZY, Fan RR, Zan X, Liang RC, Yang JL, Zhou LX, Xu JG
Title Case Report: Successful Use of BRAF/MEK Inhibitors in Aggressive BRAF-mutant Craniopharyngioma.
Journal Vol Issue Date Pages Journal Short Title
World neurosurgery 180 2023 Dec e117-e126 World Neurosurg
URL
Abstract Text Although 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.Two 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.One 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.Under specific conditions, individuals diagnosed with PCP can attain a complete tumor response following combined treatment with BRAFi/MEKi.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
BRAF V600E papillary craniopharyngioma predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical case study, neoadjuvant treatment with the combination of Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in sustained radiologic response after 3 months of treatment followed by a tumor-free period lasting 2 years from treatment discontinuation in a patient with papillary craniopharyngioma harboring BRAF V600E (PMID: 37683921). 37683921