Maciel Léa Maria Zanini, Magalhães Patrícia Künzle Ribeiro
Divisão de Endocrinologia, Departamento de Medicina Interna, Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo (FMRP-USP), Ribeirão Preto, SP, Brasil.
Arch Endocrinol Metab. 2017 Jul-Aug;61(4):398-402. doi: 10.1590/2359-3997000000267. Epub 2017 Jun 26.
Medullary thyroid carcinoma (MTC) is a rare neuroendocrine tumor originating from parafollicular C cells of the thyroid and associated with mutations in the proto-oncogene REarranged during Transfection (RET). The prognosis of MTC depends on clinical stage, with a 95.6% 10-year survival rate among patients with localized disease and 40% among patients with advanced disease. Standard chemotherapy and radiotherapy have no significant impact on the overall survival of these patients and two tyrosine kinase receptor inhibitors (TKIs), vandetanib and cabozantinib, have been recently approved for the systemic treatment of locally advanced or metastatic MTC. However, since patients with MTC and residual or recurrent disease may have an indolent course with no need for systemic treatment, and since these drugs are highly toxic, it is extremely important to select the patients who will receive these drugs in a correct manner. It is also essential to carefully monitor patients using TKI regarding possible adverse effects, which should be properly managed when occurring.
甲状腺髓样癌（MTC）是一种罕见的神经内分泌肿瘤，起源于甲状腺的滤泡旁C细胞，与转染期间重排的原癌基因（RET）突变有关。MTC的预后取决于临床分期，局限性疾病患者的10年生存率为95.6%，晚期疾病患者的10年生存率为40%。标准的化疗和放疗对这些患者的总生存期没有显著影响，两种酪氨酸激酶受体抑制剂（TKIs），凡德他尼和卡博替尼，最近已被批准用于局部晚期或转移性MTC的全身治疗。然而，由于患有MTC且有残留或复发性疾病的患者可能病程进展缓慢，无需进行全身治疗，且由于这些药物毒性很强，正确选择接受这些药物治疗的患者极为重要。密切监测使用TKI的患者是否可能出现不良反应也至关重要，不良反应发生时应妥善处理。
