索拉非尼（Sorafenib）被纳入中国医疗保险，是用于放射性碘抵抗性分化型甲状腺癌（RR-DTC）的最早的靶向药物。本研究旨在进一步证明索拉非尼在南方医科大学珠江医院用于RR-DTC的临床疗效和安全性。分析了在南方医科大学珠江医院核医学科接受治疗的RR-DTC患者（2017年10月至2020年5月）的回顾性研究。评估了用药期间的治疗效果、无进展生存期（PFS）和不良反应（AEs）。 共有31名患者被纳入研究，其中26名患者进行了有效性评估，随访时间中位数为17.5个月（4.0-51.0个月）。疾病控制率（DCR）为57.7%（n=15），客观缓解率（ORR）为26.9%（n=7）。大多数疾病控制患者甲状腺球蛋白下降超过60%（p=0.004），肺转移和仅肺转移患者的ORR较佳（分别为p=0.010和0.001）。分析的26名患者的PFS为16.5个月（95%CI: 14.41-23.90个月）。在亚组分析中，女性、仅肺转移患者、手足皮肤综合征（HFS）和甲状腺球蛋白反应≥60%的患者观察到更长的PFS（分别为p=0.038、0.045、0.035和0.000），而骨转移患者的PFS较低（p=0.035）。最常见的毒副反应是HFS（93.5%），其次是腹泻（83.9%）、脱发（74.2%）。所有副作用主要为1-2级。腹泻和HFS的3-4级不良反应比例较高。 索拉非尼在RR-DTC中显示出良好的疗效，特别是在肺转移和仅肺转移患者中。索拉非尼的不良反应一般为轻度，主要不良反应为HFS。 版权所有© 2023 Ling, Xiong, Luo, Zou, Chen, Pan, Long, Feng和Ouyang。

Sorafenib included in Chinese medical insurance is the earliest targeted drug for radioactive iodine refractory differentiated thyroid cancer (RR-DTC). This study is to further demonstrate the clinical efficacy and safety of sorafenib used in Zhujiang Hospital of Southern Medical University.RR-DTC patients treated at our Department of Nuclear Medicine in Zhujiang Hospital of Southern Medical University (October 2017-May 2020) were retrospectively analyzed. Treatment effects, progression-free survival (PFS), and adverse effects (AEs) during medication were evaluated.Of the 31 patients included, 26 patients were evaluated for efficacy with a median follow-up time of 17.5 months (4.0-51.0 months). The disease control rate (DCR) was 57.7% (n = 15) and the objective response rate (ORR) was 26.9% (n = 7). Most patients with disease control had thyroglobulin decreases of more than 60% (p = 0.004), ORRs were favorable in patients with lung metastasis and lung-only metastasis (p = 0.010 and 0.001, respectively). The PFS of the 26 patients analyzed was 16.5 months (95%CI: 14.41 -23.90 months). In the subgroup analysis, female, patients with lung-only metastasis, hand-foot skin syndrome (HFS), and thyroglobulin response ≥ 60% observed longer PFS (p = 0.038, 0.045, 0.035, and 0.000, respectively), while patients with bone metastasis had lower PFS (p = 0.035). The most common toxicity profile was HFS (93.5%), followed by diarrhea (83.9%), alopecia (74.2%). All the side effects were mainly grade 1-2. Grade 3-4 adverse reactions were more common in diarrhea and HFS.Sorafenib has promising efficacy in RR-DTC, especially in patients with lung metastasis and lung-only metastasis. The AEs of sorafenib were generally mild, and the main AE was HFS.Copyright © 2023 Ling, Xiong, Luo, Zou, Chen, Pan, Long, Feng and Ouyang.