Department of Nuclear Medicine and Endocrine Oncology, Institut Gustave Roussy, Université Paris-Sud, 114 Rue Edouard Vaillant, 94805 Villejuif, France.
Eur J Endocrinol. 2014 Mar 8;170(4):575-82. doi: 10.1530/EJE-13-0825. Print 2014 Apr.
Tyrosine kinase inhibitors (TKIs) are used to treat patients with advanced thyroid cancers. We retrospectively investigated the efficacy of TKIs administered outside of clinical trials in metastatic sites or locally advanced thyroid cancer patients from five French oncology centers.
THERE WERE 62 PATIENTS (37 MEN, MEAN AGE: 61 years) treated with sorafenib (62%), sunitinib (22%), and vandetanib (16%) outside of clinical trials; 22 had papillary, five had follicular, five had Hürthle cell, 13 had poorly differentiated, and 17 had medullary thyroid carcinoma (MTC). Thirty-three, 25, and four patients were treated with one, two, and three lines of TKIs respectively. Primary endpoints were objective tumor response rate and progression-free survival (PFS). Sequential treatments and tumor response according to metastatic sites were secondary endpoints.
Among the 39 sorafenib and 12 sunitinib treatments in differentiated thyroid carcinoma (DTC) patients, partial response (PR) rate was 15 and 8% respectively. In the 11 MTC patients treated with vandetanib, 36% had PR. Median PFS was similar in second-line compared with first-line sorafenib or sunitinib therapy (6.7 vs 7.0 months) in DTC patients, but there was no PR with second- and third-line treatments. Bone and pleural lesions were the most refractory sites to treatment.
This is the largest retrospective study evaluating TKI therapies outside of clinical trials. DTC patients treated with second-line therapy had stable disease as best response, but had a similar median PFS compared with the first-line treatment.
酪氨酸激酶抑制剂（TKIs）用于治疗晚期甲状腺癌患者。我们回顾性研究了来自五个法国肿瘤中心的转移性或局部晚期甲状腺癌患者在临床试验之外使用 TKI 的疗效。
共有 62 名患者（37 名男性，平均年龄：61 岁）在临床试验之外接受索拉非尼（62%）、舒尼替尼（22%）和凡德他尼（16%）治疗；22 名患者患有乳头状癌，5 名患者患有滤泡状癌，5 名患者患有 Hurthle 细胞癌，13 名患者患有低分化癌，17 名患者患有甲状腺髓样癌（MTC）。33、25 和 4 名患者分别接受了一线、二线和三线 TKI 治疗。主要终点是客观肿瘤缓解率和无进展生存期（PFS）。次要终点是按转移部位的序贯治疗和肿瘤反应。
在分化型甲状腺癌（DTC）患者中，39 例索拉非尼和 12 例舒尼替尼治疗中，部分缓解（PR）率分别为 15%和 8%。在接受凡德他尼治疗的 11 例 MTC 患者中，36%有 PR。在 DTC 患者中，二线索拉非尼或舒尼替尼治疗与一线治疗相比，中位 PFS 相似（6.7 与 7.0 个月），但二线和三线治疗无 PR。骨和胸膜病变是对治疗最具耐药性的部位。
这是评估临床试验之外 TKI 治疗的最大回顾性研究。二线治疗的 DTC 患者最佳反应为疾病稳定，但与一线治疗相比，中位 PFS 相似。
