Beneficial effects of vemurafenib on craniopharyngioma carrying BRAF-V600E mutation
10.16352/j.issn.1001-6325.2024.06.0866
- VernacularTitle:维莫非尼有效治疗BRAF-V600E突变所致的难治性颅咽管瘤
- Author:
Xi WANG
1
;
Ting YE
;
Min NIE
;
Xueyan WU
;
Jiangfeng MAO
Author Information
1. 中国医学科学院 北京协和医学院 北京协和医院 内分泌科,北京 100730
- Keywords:
papillary craniopharyngioma;
BRAF-V600E mutation;
BRAF inhibitors;
MEK inhibitors
- From:
Basic & Clinical Medicine
2024;44(6):866-872
- CountryChina
- Language:Chinese
-
Abstract:
Objective To evaluate the efficacy and adverse reactions of BRAF inhibitor vermorafenib on the treat-ment 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.Lit-erature 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)Litera-ture 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.
