Division of Hematology/Oncology, Department of Medicine, Abramson Cancer Center, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Department of Pathology and Laboratory Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Eur Thyroid J. 2024 Nov 20;13(6). doi: 10.1530/ETJ-24-0225. Print 2024 Dec 1.
Poorly differentiated thyroid carcinomas (PDTCs) are rare and aggressive head and neck malignancies with a poor prognosis. Systemic treatment for incurable PDTC consists of multi-kinase inhibitors (MKIs) based on extrapolation from the experience with radioiodine refractory differentiated thyroid cancer (DTC). Cabozantinib is an approved second-line MKI therapy for DTC, but there are limited data regarding the safety and efficacy of cabozantinib for PDTC.
We conducted a single-institution, retrospective analysis of patients with PDTC who received cabozantinib in any line of therapy. Baseline demographics, disease characteristics, treatment history, toxicity, and clinical outcomes were abstracted from the electronic medical record. Median progression-free survival (PFS) and overall survival (OS) were primary endpoints and estimated using Kaplan-Meier methodology.
Seven patients with PDTC who received cabozantinib were included. 4/7 (57%) patients had a partial response to cabozantinib, while 2/7 (29%) had stable disease (SD) as their best response. The median time on treatment for cabozantinib was 10.53 months. The median PFS from the start of cabozantinib was 12.9 months, and median OS was 14.21 months. Most adverse events to treatment (5/6) were low grade. Two (29%) patients were alive at the date of the last follow-up.
Cabozantinib is an effective and reasonably well-tolerated treatment option for patients with PDTC. Prospective studies are needed to further investigate the role of cabozantinib in the treatment of PDTC, alone and in combination with other agents, including checkpoint inhibitors.
未分化甲状腺癌（PDTC）是一种罕见且侵袭性的头颈部恶性肿瘤，预后较差。对于无法治愈的 PDTC，系统治疗包括多激酶抑制剂（MKIs），这是基于放射性碘难治性分化型甲状腺癌（DTC）的经验推断而来。卡博替尼是一种获批的 DTC 二线 MKI 治疗药物，但关于卡博替尼治疗 PDTC 的安全性和疗效的数据有限。
我们对接受卡博替尼任何线治疗的 PDTC 患者进行了单中心回顾性分析。从电子病历中提取基线人口统计学、疾病特征、治疗史、毒性和临床结局等数据。无进展生存期（PFS）和总生存期（OS）的中位数是主要终点，使用 Kaplan-Meier 方法进行估计。
纳入了 7 名接受卡博替尼治疗的 PDTC 患者。4/7（57%）患者对卡博替尼有部分缓解，而 2/7（29%）患者的最佳反应为疾病稳定（SD）。卡博替尼的中位治疗时间为 10.53 个月。从开始使用卡博替尼到疾病进展的中位 PFS 为 12.9 个月，中位 OS 为 14.21 个月。大多数治疗相关不良反应（5/6）为低级别。截至最后一次随访时，有 2 名（29%）患者存活。
卡博替尼是 PDTC 患者有效且耐受性良好的治疗选择。需要前瞻性研究进一步探讨卡博替尼单独或与其他药物（包括检查点抑制剂）联合治疗 PDTC 的作用。
