BRAF 状态可能预测对伊马替尼、舒尼替尼和瑞戈非尼耐药的胃肠道间质瘤对索拉非尼的反应：病例系列和文献复习。
The BRAF Status May Predict Response to Sorafenib in Gastrointestinal Stromal Tumors Resistant to Imatinib, Sunitinib, and Regorafenib: Case Series and Review of the Literature.
机构信息
Department of Gastroenterology, Hepatology and Infectious Diseases, Magdeburg, Germany.
Department of Pathology, Otto-von-Guericke University Hospital, Magdeburg, Germany.
出版信息
Digestion. 2019;99(2):179-184. doi: 10.1159/000490886. Epub 2018 Sep 4.
BACKGROUND
Sorafenib has shown efficacy in patients with imatinib-, sunitinib-, and regorafenib-resistant gastrointestinal stromal tumors (GISTs). No biomarker is currently available for predicting response to sorafenib in patients with GIST.
METHODS
We herein report 3 patients with imatinib-, sunitinib-, and regorafenib-resistant metastasized GISTs, who were treated with sorafenib. Besides receptor tyrosine kinase KIT and platelet-derived growth factor receptor α, also BRAF was tested for mutations.
RESULTS
Sorafenib therapy induced a long-term disease control in 2 out of 3 patients over a period of 49 and 19 months, respectively. Sorafenib-responsive GISTs were BRAF wild-type, whereas the sorafenib-resistant GIST carried a BRAF V600E mutation.
CONCLUSION
We confirm sorafenib as an effective therapeutic option in patients with imatinib-, sunitinib-, and regorafenib-resistant GISTs. Larger studies are required to corroborate whether BRAF mutation may predict sorafenib resistance in GISTs.
背景
索拉非尼已显示出对伊马替尼、舒尼替尼和regorafenib 耐药的胃肠道间质瘤（GIST）患者的疗效。目前尚无预测 GIST 患者对索拉非尼反应的生物标志物。
方法
我们在此报告 3 例伊马替尼、舒尼替尼和regorafenib 耐药转移性 GIST 患者，他们接受了索拉非尼治疗。除了受体酪氨酸激酶 KIT 和血小板衍生生长因子受体 α 外，还检测了 BRAF 突变情况。
结果
索拉非尼治疗在 3 例患者中的 2 例中诱导了长达 49 个月和 19 个月的长期疾病控制。索拉非尼应答的 GIST 为 BRAF 野生型，而索拉非尼耐药的 GIST 携带 BRAF V600E 突变。
结论
我们证实索拉非尼是伊马替尼、舒尼替尼和regorafenib 耐药 GIST 患者的有效治疗选择。需要更大规模的研究来证实 BRAF 突变是否可以预测 GIST 对索拉非尼的耐药性。
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