Section of Endocrinology, Diabetes and Metabolism, Department of Medicine, University of Chicago, 5841 S. Maryland Ave., Chicago, IL 60645, USA.
Adv Ther. 2012 Nov;29(11):925-34. doi: 10.1007/s12325-012-0060-6. Epub 2012 Oct 25.
Patients with advanced medullary thyroid cancer (MTC) have poor prognoses and limited treatment options. Improved knowledge about molecular aberrations associated with MTC and the availability of novel targeted tyrosine kinase inhibitors (TKIs) have led to new potential treatment modalities. Cabozantinib is an oral multitargeted TKI with activity against multiple receptors including RET, vascular endothelial growth factor receptor type 2 (VEGFR2), and MET that has been evaluated in MTC in the preclinical and clinical arenas.
This article reviews unmet clinical needs in advanced MTC. The authors consider novel agents that have been studied in MTC, with a focus on the investigational agent cabozantinib. Up-to-date clinical data of cabozantinib in MTC are discussed.
Recent clinical evaluation suggests that cabozantinib is the first agent to prolong progression-free survival in patients with progressive MTC. These findings indicate that cabozantinib may be an effective therapy in advanced MTC. No improvement in overall survival has been demonstrated but data are not mature.
Cabozantinib may be an effective treatment option for patients with advanced MTC and is worthy of further evaluation.
晚期甲状腺髓样癌（MTC）患者预后较差，治疗选择有限。对与 MTC 相关的分子异常的认识提高，以及新型靶向酪氨酸激酶抑制剂（TKI）的出现，为新的潜在治疗方法提供了可能。卡博替尼是一种口服多靶点 TKI，对包括 RET、血管内皮生长因子受体 2（VEGFR2）和 MET 在内的多种受体具有活性，已在临床前和临床领域对 MTC 进行了评估。
本文综述了晚期 MTC 未满足的临床需求。作者考虑了已在 MTC 中研究过的新型药物，重点介绍了研究中的卡博替尼。讨论了卡博替尼在 MTC 中的最新临床数据。
最近的临床评估表明，卡博替尼是首个延长进展性 MTC 患者无进展生存期的药物。这些发现表明卡博替尼可能是晚期 MTC 的有效治疗方法。虽然未显示总生存期的改善，但数据尚不成熟。
卡博替尼可能是晚期 MTC 患者的有效治疗选择，值得进一步评估。
