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病例报告和文献复习:BRAF-V600 抑制剂治疗颅咽管瘤:一种潜在的治疗范式转变。

Case report and literature review of BRAF-V600 inhibitors for treatment of papillary craniopharyngiomas: A potential treatment paradigm shift.

机构信息

Department of Neurosurgery, National Brain Aneurysm & Tumor Center, United Hospital, St. Paul, MN, USA.

Midwest Radiology, St. Paul, MN, USA.

出版信息

J Clin Pharm Ther. 2022 Jun;47(6):826-831. doi: 10.1111/jcpt.13600. Epub 2022 Jan 12.

Abstract

WHAT IS KNOWN AND OBJECTIVE

The BRAF-V600E genetic mutation offers a potential targeted therapy for the treatment of papillary craniopharyngiomas.

CASE SUMMARY

A 35-year-old man underwent a craniotomy and subtotal resection of a large BRAF-V600E-positive papillary craniopharyngioma before referral to our institution. Our treatment included the BRAF-V600 inhibitor dabrafenib mesylate (75 mg, twice/day) and trametinib dimethyl sulfoxide (2 mg/day). The residual tumour decreased in size by 95% over 21 months without negative side effects.

WHAT IS NEW AND CONCLUSION

We reviewed the literature on BRAF-V600E inhibition as a non-invasive method of treating papillary craniopharyngiomas harbouring the BRAF-V600E mutation.

摘要

已知和目的

BRAF-V600E 基因突变提供了一种针对治疗颅咽管瘤的潜在靶向治疗方法。

病例总结

一名 35 岁男性在转至我院之前接受了颅咽管瘤的开颅手术和次全切除术,肿瘤呈 BRAF-V600E 阳性。我们的治疗包括 BRAF-V600 抑制剂甲磺酸达拉非尼(75mg,每日两次)和曲美替尼二甲亚砜(2mg,每日一次)。在 21 个月内,残余肿瘤缩小了 95%,没有出现负面副作用。

新发现和结论

我们回顾了 BRAF-V600E 抑制作为治疗携带 BRAF-V600E 突变的颅咽管瘤的非侵入性方法的文献。

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