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靶向治疗携带 BRAF V600E 突变的颅咽管瘤。

Targeted treatment of papillary craniopharyngiomas harboring BRAF V600E mutations.

机构信息

Department of Neurosurgery, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.

Divisions of Neuro-Oncology and Hematology/Oncology, Departments of Neurology and Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.

出版信息

Cancer. 2019 Sep 1;125(17):2910-2914. doi: 10.1002/cncr.32197. Epub 2019 Jul 17.

Abstract

Papillary craniopharyngiomas (PCPs) are characterized by the presence of BRAF V600E mutations, which are emerging as a useful guide for diagnosis and treatment decision making. The ongoing multicenter phase 2 Alliance A071601 trial is evaluating the efficacy of BRAF and mitogen-activated protein kinase kinase (MEK) inhibitors for patients with PCPs. With continued successful responses, it is proposed that BRAF (and MEK) inhibitors be evaluated for the neoadjuvant treatment of patients with PCPs.

摘要

乳头型颅咽管瘤(PCP)的特征是存在 BRAF V600E 突变,该突变正成为诊断和治疗决策的有用指导。正在进行的多中心联盟 A071601 二期试验正在评估 BRAF 和丝裂原活化蛋白激酶激酶(MEK)抑制剂对 PCP 患者的疗效。随着持续成功的反应,提议评估 BRAF(和 MEK)抑制剂作为 PCP 患者的新辅助治疗。

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