Department of Endocrine Surgery, University of California, San Francisco, San Francisco, California, USA.
Curr Opin Oncol. 2013 Jan;25(1):39-43. doi: 10.1097/CCO.0b013e32835a42b9.
Vandetanib is a small molecule tyrosine kinase inhibitor that has been recently approved as an 'orphan drug' for the treatment of patients with unresectable, locally advanced, or metastatic medullary thyroid cancer (MTC).
MTC is a neuroendocrine malignancy frequently associated with mutations to the RET proto-oncogene. Vandetanib selectively targets RET, vascular endothelial growth factor receptor-2, and epidermal growth factor receptor dependent signaling. Vandetanib has been shown to improve progression-free survival in patients with advanced MTC. In general, vandetanib is well tolerated, but QTc prolongation remains a potential concern demanding careful patient selection and monitoring.
Vandetanib has emerged as one of the more promising small molecule tyrosinse kinase inhibitors, providing durable rates of disease stabilization, with an acceptable adverse event profile in patients with advanced MTC.
凡德他尼是一种小分子酪氨酸激酶抑制剂，最近被批准为治疗无法切除的局部晚期或转移性甲状腺髓样癌（MTC）患者的“孤儿药”。
MTC 是一种神经内分泌恶性肿瘤，常与 RET 原癌基因的突变有关。凡德他尼选择性地针对 RET、血管内皮生长因子受体-2 和表皮生长因子受体依赖性信号通路。凡德他尼已被证明可改善晚期 MTC 患者的无进展生存期。一般来说，凡德他尼耐受性良好，但 QTc 延长仍然是一个潜在的问题，需要仔细选择患者并进行监测。
凡德他尼已成为更有前途的小分子酪氨酸激酶抑制剂之一，为晚期 MTC 患者提供了持久的疾病稳定率，且具有可接受的不良事件谱。
