Jichu yixue yu linchuang (Jun 2024)

Beneficial effects of vemurafenib on craniopharyngioma carrying BRAF-V600E mutation

  • WANG Xi, YE Ting, NIE Min, WU Xueyan, MAO Jiangfeng

DOI
https://doi.org/10.16352/j.issn.1001-6325.2024.06.0866
Journal volume & issue
Vol. 44, no. 6
pp. 866 – 872

Abstract

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Objective To evaluate the efficacy and adverse reactions of BRAF inhibitor vermorafenib on the treatment of refractory craniopharyngioma carrying BRAF-V600E mutation. Methods Clinical data of two patients with refractory craniopharyngiomas (CP) were recorded and reviewed. The patients were followed up for 3-5 years. Literature on CPs receiving BRAF or BRAF/MEK therapy was reviewed. Results 1)Papillary CP progressed after multiple operations and radiotherapy in two patients. Further treatments were very difficult. 2)The presence of BRAF-V600E mutation in the tumor was confirmed, and vermorafenib was administered for 6.5-7.5 months. Tumor volumes remarkably shrank by 95%-99%. No tumor relapse was observed during the follow-up of 3-5 years after discontinuation of vemurafenib. 3)The main adverse reaction was rash, which was dose dependent. 4)Literature review showed the volume shrank by 50%-100% in 33/34 tumors during BRAF or BRAF/MEK inhibitor therapy. Conclusions BRAF inhibitor vemurafenib is effective in treating refractory craniopharyngioma carrying BRAF-V600E mutation with endurable side effects, which may bring some changes to the management of CP in future.

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