Sousa Santos Francisco, Joana Santos Rita, Leite Valeriano
Endocrinology Department, Centro Hospitalar Lisboa Ocidental, Lisbon, Portugal.
Endocrinology Department, Instituto Português de Oncologia de Lisboa Francisco Gentil, Lisbon, Portugal.
Eur Thyroid J. 2019 Oct;8(5):262-267. doi: 10.1159/000501680. Epub 2019 Aug 15.
Radioactive iodine (RAI)-refractory differentiated thyroid cancer (DTC) is a rare form of DTC which poses a therapeutic challenge due to the scarcity of effective treatment options. In recent years several tyrosine kinase inhibitors targeting specific molecular pathways involved in its pathogenesis have been investigated, such as sorafenib, lenvatinib, and sunitinib. These appear to be associated with improved progression-free survival (PFS).
We aim to describe our experience with sorafenib and sunitinib in the treatment of RAI-refractory metastatic DTC and to evaluate and compare their efficacy and adverse effect profiles.
A total of 28 patients with RAI-refractory metastatic DTC were included - 26 had first-line treatment with sorafenib (8 subsequently switched to sunitinib, most due to disease progression) and 2 with sunitinib. We evaluated PFS and best radiological response achieved with each agent as primary endpoints. The secondary objective was to describe adverse effects and safety profile.
Mean PFS was 10.8 months with sorafenib and 6 months with sunitinib as a second-line treatment. Best overall response was partial remission (PR) with either agent - PR rate of 30.7% with sorafenib and 37.5% with second-line sunitinib. All treatment courses had registered adverse effects and 13.9% justified definitive treatment cessation.
Sorafenib and sunitinib appear to be effective treatment options in delaying disease progression of patients with RAI-refractory metastatic DTC, with an acceptable safety profile. Interestingly, sunitinib appears to show some efficacy even in patients who experience disease progression on sorafenib.
放射性碘（RAI）难治性分化型甲状腺癌（DTC）是一种罕见的DTC形式，由于有效治疗选择稀缺，对治疗构成挑战。近年来，已经研究了几种针对其发病机制中特定分子途径的酪氨酸激酶抑制剂，如索拉非尼、仑伐替尼和舒尼替尼。这些药物似乎与无进展生存期（PFS）的改善有关。
我们旨在描述我们使用索拉非尼和舒尼替尼治疗RAI难治性转移性DTC的经验，并评估和比较它们的疗效和不良反应谱。
共纳入28例RAI难治性转移性DTC患者，26例接受索拉非尼一线治疗（8例随后改用舒尼替尼，大多数是由于疾病进展），2例接受舒尼替尼治疗。我们将每种药物的PFS和最佳放射学反应作为主要终点进行评估。次要目标是描述不良反应和安全性。
索拉非尼作为一线治疗的平均PFS为10.8个月，舒尼替尼作为二线治疗的平均PFS为6个月。两种药物的最佳总体反应均为部分缓解（PR），索拉非尼的PR率为30.7%，二线舒尼替尼的PR率为37.5%。所有治疗疗程均有不良反应记录，13.9%的患者因不良反应而停止治疗。
索拉非尼和舒尼替尼似乎是延缓RAI难治性转移性DTC患者疾病进展的有效治疗选择，安全性可接受。有趣的是，舒尼替尼似乎对在索拉非尼治疗期间出现疾病进展的患者也有一定疗效。
