Department of Nuclear Medicine, Shanghai Jiao Tong University , Shanghai, People's Republic of China.
Thyroid. 2011 Feb;21(2):119-24. doi: 10.1089/thy.2010.0199. Epub 2010 Dec 27.
Sorafenib has shown promise in the treatment of patients with advanced or metastatic thyroid carcinoma. However, the optimal dose has not been established and data on Chinese population are not available. We conducted a study to assess the responses to sorafenib at a low dose of 200 mg twice daily in patients with progressive radioiodine-refractory pulmonary metastases from papillary thyroid carcinoma (PTC).
Eligible patients received sorafenib 200 mg orally twice daily. Responses were assessed using Response Evaluation Criteria in Solid Tumors and adverse events were assessed periodically. The end points included response rate and progression-free survival.
Nine patients with radioiodine-refractory PTC were enrolled in the study and treated for a minimum of 13 weeks. The objective partial response rate was 33%. The stable disease rate was 44%. The mean progression-free survival was 42 weeks (95% confidence interval, 29.5 to 53.9). Two patients showed disease progression, and one of them died at 4 months after beginning of treatment. There was a marked and rapid change in the serum thyroglobulin level after start of treatment, with a mean decrease of 60% within 12 weeks, consistent with radiographic findings. Although the types of toxicities were consistent with other sorafenib trials, their severity was relatively mild. None of the patients discontinued sorafenib or reduced their dose because of treatment-related adverse events.
Sorafenib at a dose of 200 mg twice daily has a potential therapeutic effect and is well tolerated in Chinese patients with PTC and radioiodine-refractory pulmonary metastases. Further study is warranted with a larger cohort of patients.
索拉非尼在治疗晚期或转移性甲状腺癌患者方面显示出了一定的疗效。然而，其最佳剂量尚未确定，且目前尚无中国人群的数据。我们开展了一项研究，旨在评估低剂量（每日两次，每次 200mg）索拉非尼治疗放射性碘难治性甲状腺乳头状癌（PTC）肺转移进展患者的疗效。
符合条件的患者接受索拉非尼 200mg 口服，每日两次。采用实体瘤疗效评价标准评估应答，定期评估不良事件。主要终点包括应答率和无进展生存期。
本研究共纳入 9 例放射性碘难治性 PTC 患者，治疗时间至少为 13 周。客观部分缓解率为 33%。疾病稳定率为 44%。中位无进展生存期为 42 周（95%置信区间，29.5 至 53.9）。2 例患者出现疾病进展，其中 1 例在开始治疗后 4 个月死亡。开始治疗后，血清甲状腺球蛋白水平出现显著且快速的变化，12 周内平均下降 60%，与影像学发现一致。虽然毒性类型与其他索拉非尼试验一致，但严重程度相对较轻。无患者因治疗相关不良事件而停止索拉非尼或减少剂量。
索拉非尼每日两次、每次 200mg 的剂量在中国 PTC 伴放射性碘难治性肺转移患者中具有潜在的治疗效果，且耐受性良好。需要进一步研究，以纳入更大的患者队列。
