Zhang Yuqing, Zhang Xiaoxin, Lin Lifan, Xing Mingzhao
School of Medicine, Southern University of Science and Technology, Shenzhen, Guangdong 518055, China.
Department of Pulmonary and Critical Care Medicine, State Key Laboratory of Complex, Severe and Rare Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China.
J Clin Endocrinol Metab. 2025 Feb 18;110(3):873-886. doi: 10.1210/clinem/dgae617.
There has been considerable success in the development of drugs for targeted therapy of radioiodine-refractory differentiated thyroid cancer (RR-DTC) and to know the safety and efficacy of these drugs will help their appropriate application.
To evaluate the efficacy and safety of current targeted drug therapies for radioiodine-refractory differentiated thyroid cancer.
This was a meta-analysis of relevant randomized controlled trials (RCTs) and single-arm studies searched across PubMed, Embase, Cochranes, and Web of Sciences up to September 12, 2023. Stata15.0 software was used to assess overall survival (OS), progression-free survival (PFS), disease control rate (DCR), objective response rate (ORR), and adverse events. The Cochrane Bias Risk tool was used to assess literature quality and trial bias and RevMan 5.4 was used to generate a quality assessment map.
A total of 8 RCTs and 17 single-arm studies with 3270 patients on 7 drugs-vandetanib, sorafenib, lenvatinib, cabozantinib, apatinib, donafenib, and anlotinib-were included. Targeted therapy with these drugs effectively prolonged PFS and OS in patients with RR-DTC with overall hazard ratios of 0.35 (95% CI 0.23-0.53, P < .00001) and 0.53 (95% CI 0.32-0.86, P < .00001), respectively. ORR and DCR were also prolonged, with overall risk ratios of 27.63 (95% CI 12.39-61.61, P < .00001) and 1.66 (95% CI 1.48-1.86, P < .00001), respectively. The subgroup analysis using effect size (ES) showed that apatinib had the best effect on ORR with an ES of 0.66 (95% CI 0.49-0.83, P < .00001) and DCR with a ES of 0.95 (95% CI 0.91-1.00, P < .00001). Common drug adverse events included hypertension, diarrhea, proteinuria, and fatigue.
The currently used targeted drug therapies for RR-DTC can significantly improve clinical outcomes, and the new drug apatinib demonstrates promise for potentially superior performance.
放射性碘难治性分化型甲状腺癌（RR-DTC）的靶向治疗药物研发取得了显著成功，了解这些药物的安全性和有效性将有助于其合理应用。
评估目前用于放射性碘难治性分化型甲状腺癌的靶向药物治疗的疗效和安全性。
这是一项对相关随机对照试验（RCT）和单臂研究的荟萃分析，检索了截至2023年9月12日的PubMed、Embase、Cochranes和Web of Sciences数据库。使用Stata15.0软件评估总生存期（OS）、无进展生存期（PFS）、疾病控制率（DCR）、客观缓解率（ORR）和不良事件。使用Cochrane偏倚风险工具评估文献质量和试验偏倚，并使用RevMan 5.4生成质量评估图。
共纳入8项RCT和17项单臂研究，涉及3270例使用7种药物（凡德他尼、索拉非尼、乐伐替尼、卡博替尼、阿帕替尼、多纳非尼和安罗替尼）的患者。这些药物的靶向治疗有效延长了RR-DTC患者的PFS和OS，总体风险比分别为0.35（95%CI 0.23-0.53，P<.00001）和0.53（95%CI 0.32-0.86，P<.00001）。ORR和DCR也得到延长，总体风险比分别为27.63（95%CI 12.39-61.61，P<.00001）和1.66（95%CI 1.48-1.86，P<.00001）。使用效应量（ES）的亚组分析表明，阿帕替尼对ORR的效果最佳，ES为0.66（95%CI 0.49-0.83，P<.00001），对DCR的效果最佳，ES为0.95（95%CI 0.91-1.00，P<.00001）。常见的药物不良事件包括高血压、腹泻、蛋白尿和疲劳。
目前用于RR-DTC的靶向药物治疗可显著改善临床结局，新药阿帕替尼显示出潜在的优越性能。
