Hong David, Ye Lei, Gagel Robert, Chintala Lakshmi, El Naggar Adel K, Wright John, Kurzrock Razelle
Department of Investigational Therapeutics (Phase I Program), Unit 455, Division of Cancer Medicine, M. D. Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Mol Cancer Ther. 2008 May;7(5):1001-6. doi: 10.1158/1535-7163.MCT-07-2422. Epub 2008 Apr 29.
Medullary thyroid carcinoma (MTC) is an uncommon malignancy of hereditary and sporadic presentation. Mutations in the RET proto-oncogene are involved in the pathogenesis of familial MTC and >50% of the sporadic cases. Currently, there is no effective treatment for recurrent or metastatic MTC. We report here a rapid response to a sorafenib (RET and RAF kinase and vascular endothelial growth factor receptor inhibitor)--based regimen in a patient with sporadic MTC who had advanced, progressive disease and a novel RET kinase aberration at exon 11 shown in tumor tissue.
甲状腺髓样癌（MTC）是一种具有遗传性和散发性表现的罕见恶性肿瘤。RET原癌基因的突变参与了家族性MTC和超过50%的散发性病例的发病机制。目前，对于复发性或转移性MTC尚无有效的治疗方法。我们在此报告了一名散发性MTC患者对基于索拉非尼（RET和RAF激酶以及血管内皮生长因子受体抑制剂）的治疗方案产生快速反应，该患者患有晚期进行性疾病，且肿瘤组织显示第11外显子存在一种新的RET激酶异常。
