1 Division of Endocrinology and Metabolism, Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine , Seoul, Korea.
2 Division of Endocrinology and Metabolism, Department of Medicine, Thyroid Center, Samsung Medical Center, Sungkyunkwan University School of Medicine , Seoul, Korea.
Thyroid. 2018 Mar;28(3):340-348. doi: 10.1089/thy.2017.0356. Epub 2018 Feb 16.
Sorafenib, a multi-kinase inhibitor, is approved for the treatment of patients with radioactive iodine (RAI)-refractory differentiated thyroid cancer (DTC). This study evaluated the efficacy and safety of sorafenib in real-world clinical practice and compared the results to those of the DECISION trial. The clinical features associated with better clinical outcomes after sorafenib treatment were also evaluated.
This multicenter, retrospective cohort study evaluated 98 patients with progressive RAI-refractory DTC who were treated with sorafenib in six tertiary hospitals in Korea. The primary objective was the progression-free survival (PFS) according to Response Evaluation Criteria In Solid Tumors v1.1. Overall survival, response rate (defined as the best objective response according to Response Evaluation Criteria In Solid Tumors v1.1), and safety were also evaluated.
The median PFS was 9.7 months; median overall survival was not reached during follow-up. Partial responses and stable disease were achieved in 25 (25%) and 64 (65%) patients, respectively. Stable disease of >6 months was achieved in 41 (42%) patients. Subgroup analyses identified several prognostic indicators of a better PFS: absence of disease-related symptoms (hazard ratio [HR] = 0.5; p = 0.041), lung-only metastasis (HR = 0.4; p = 0.048), a daily maintenance dose ≥600 mg (HR = 0.3; p = 0.005), and a thyroglobulin reduction ≥60% (HR = 0.4; p = 0.012). The mean daily dose of sorafenib was 666 ± 114 mg, and drug withdrawals due to adverse events (AEs) occurred in 13% of patients. AEs and serious AEs were reported in 93 (95%) and 40 (41%) patients, respectively. The most frequent AE was hand-foot skin reaction (76%).
The PFS of progressive RAI-refractory DTC patients treated with sorafenib was consistent with the findings of the DECISION trial. Disease-related symptoms, lung-only metastasis, a daily maintenance dose, and thyroglobulin reduction were significantly associated with PFS. These results suggest that sorafenib is an effective treatment option for patients with progressive RAI-refractory DTC.
索拉非尼是一种多激酶抑制剂，已被批准用于治疗放射性碘（RAI）难治性分化型甲状腺癌（DTC）患者。本研究评估了索拉非尼在真实临床实践中的疗效和安全性，并将结果与 DECISION 试验进行了比较。还评估了索拉非尼治疗后临床结局更好的临床特征。
这是一项多中心、回顾性队列研究，评估了 98 例在韩国 6 家三级医院接受索拉非尼治疗的进展性 RAI 难治性 DTC 患者。主要终点是根据实体瘤反应评价标准 1.1 版评估的无进展生存期（PFS）。还评估了总生存期、客观缓解率（根据实体瘤反应评价标准 1.1 版评估的最佳客观缓解定义）和安全性。
中位 PFS 为 9.7 个月；中位总生存期在随访期间未达到。25 例（25%）和 64 例（65%）患者分别达到部分缓解和疾病稳定。41 例（42%）患者疾病稳定时间超过 6 个月。亚组分析确定了几个与更好的 PFS 相关的预后指标：无疾病相关症状（风险比 [HR] = 0.5；p = 0.041）、仅肺转移（HR = 0.4；p = 0.048）、每日维持剂量≥600mg（HR = 0.3；p = 0.005）和甲状腺球蛋白降低≥60%（HR = 0.4；p = 0.012）。索拉非尼的平均日剂量为 666±114mg，因不良反应（AE）而停药的患者占 13%。93 例（95%）和 40 例（41%）患者报告了 AE 和严重 AE。最常见的 AE 是手足皮肤反应（76%）。
接受索拉非尼治疗的进展性 RAI 难治性 DTC 患者的 PFS 与 DECISION 试验结果一致。疾病相关症状、仅肺转移、每日维持剂量和甲状腺球蛋白降低与 PFS 显著相关。这些结果表明，索拉非尼是治疗进展性 RAI 难治性 DTC 患者的有效治疗选择。
