1 Department of Medical Oncology, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital , Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China .
2 Key Laboratory of Carcinogenesis and Translational Research (Ministry of Education/Beijing), The VIPII Gastrointestinal Cancer Division of Medical Department, Peking University Cancer Hospital and Institute , Beijing, China .
Thyroid. 2018 Nov;28(11):1455-1461. doi: 10.1089/thy.2018.0022. Epub 2018 Oct 26.
The prognosis of advanced or metastatic medullary thyroid carcinoma (MTC) is poor, and there are few therapeutic options. Anlotinib has previously shown promising antitumor activity on MTC in preclinical models and a Phase I study. This Phase II clinical trial was devised to confirm the antitumor activity of anlotinib in patients with advanced or metastatic MTC.
Patients with unresectable locally advanced or metastatic MTC received once daily oral anlotinib 12 mg, two weeks on/one week off, until disease progression, death, unacceptable toxicity, or withdrawal of consent for any reason. The dose was adjusted on the basis of observed toxicity. The primary endpoint was progression-free survival (PFS).
Fifty-eight patients received anlotinib treatment. The primary endpoint PFS has not yet been reached at the time of analysis. On the basis of investigator assessments, 56.9% of patients experienced a partial response. PFS rate at 48 weeks was 85.5%. Forty-five patients had a ≥50% decrease in serum calcitonin concentration from baseline. The most common adverse events were hand-foot syndrome, hypertriglyceridemia, cholesterol elevation, fatigue, and proteinuria.
Anlotinib demonstrated a durable antitumor activity with a manageable adverse event profile in locally advanced or metastatic MTC.
晚期或转移性甲状腺髓样癌（MTC）的预后较差，治疗选择有限。安罗替尼先前在临床前模型和 I 期研究中显示出对 MTC 的有前景的抗肿瘤活性。本 II 期临床试验旨在确认安罗替尼在晚期或转移性 MTC 患者中的抗肿瘤活性。
不可切除的局部晚期或转移性 MTC 患者接受每日一次口服安罗替尼 12mg，每两周一次/一周一次，直到疾病进展、死亡、无法耐受的毒性或因任何原因撤回同意。根据观察到的毒性调整剂量。主要终点是无进展生存期（PFS）。
58 名患者接受了安罗替尼治疗。分析时主要终点 PFS 尚未达到。根据研究者评估，56.9%的患者出现部分缓解。48 周时的 PFS 率为 85.5%。45 名患者的血清降钙素浓度从基线下降了≥50%。最常见的不良反应是手足综合征、甘油三酯升高、胆固醇升高、疲劳和蛋白尿。
安罗替尼在局部晚期或转移性 MTC 中表现出持久的抗肿瘤活性，且具有可管理的不良事件谱。
