Background:Meta-analyses aimed to assess the effectiveness and safety of targeted and contemporary therapies utilised in locally advanced and metastatic anaplastic thyroid cancer (ATC).Methods:Employing PRISMA and MOOSE guidelines, PubMed, Scopus, Cochrane Library and Web of Science were explored from the inception of targeted therapy until December 2024. A meta-analysis was performed to evaluate the effectiveness, toxicity and survival outcomes of various mutationally directed agents, chemotherapy and radiotherapy in locally advanced/metastatic ATC cases.Results:A total of 47 studies (26 prospective phase II trials and 21 retrospective studies) involving 980 patients met the inclusion criteria. The pooled results showed an overall response rate (ORR) of 29.7% (95% CI: 25.4–34.2%;I2= 42.4%;p< 0.0001). A total of 49.9% deaths were reported, although a significant number remained alive compared to baseline (mean difference [MD]: 2.07, 95% CI: 1.90–2.24;I2= 88.6%;p< 0.0001). The pooled median progression-free survival (PFS) was 5.4 months (95% CI: 4.0–6.7 months;I2= 97.9%;p< 0.0001). Dabrafenib/trametinib (DT) with and without pembrolizumab and lenvatinib plus pembrolizumab (LP) were associated with higher ORR rates and improved OS and PFS. About 51.% of studies mentioned bio-marker analysis (BRAFV600[14.7%],PDL1[9.2%],RAS[1.1%],PIK3CA[1.0%] andNTRK1/3[0.7%]). Toxicity was reported in 94.7% of patients.Conclusions:This meta-analysis found that DT could be a promising first-line treatment option forBRAFV600-mutated ATC, with or without immunotherapy. Alternatively, LP shows potential inBRAFV600wild-type andPDL1-overexpressing cases. Routine biomarker analysis remains critical for optimising ATC management strategies.
背景：本研究旨在通过荟萃分析评估局部晚期及转移性间变性甲状腺癌（ATC）中靶向治疗与当代疗法的有效性与安全性。 方法：遵循PRISMA与MOOSE指南，系统检索了自靶向疗法问世至2024年12月期间PubMed、Scopus、Cochrane Library及Web of Science数据库。通过荟萃分析评估局部晚期/转移性ATC病例中各类突变导向药物、化疗及放疗的疗效、毒性和生存结局。 结果：共纳入47项研究（26项前瞻性II期试验和21项回顾性研究），涉及980例患者。汇总分析显示总体缓解率（ORR）为29.7%（95% CI：25.4–34.2%；I²=42.4%；p<0.0001）。尽管相较于基线有显著数量的患者存活（均数差[MD]：2.07，95% CI：1.90–2.24；I²=88.6%；p<0.0001），但总死亡率为49.9%。汇总中位无进展生存期（PFS）为5.4个月（95% CI：4.0–6.7个月；I²=97.9%；p<0.0001）。达拉非尼/曲美替尼（DT）联合或不联合帕博利珠单抗，以及仑伐替尼联合帕博利珠单抗（LP）方案与更高的ORR及改善的总生存期（OS）和PFS相关。约51%的研究提及生物标志物分析（BRAFV600[14.7%]、PDL1[9.2%]、RAS[1.1%]、PIK3CA[1.0%]及NTRK1/3[0.7%]）。94.7%的患者报告了治疗相关毒性。 结论：本荟萃分析表明，对于BRAFV600突变的ATC，无论是否联合免疫治疗，DT均可能成为有前景的一线治疗方案。此外，LP在BRAFV600野生型且PDL1过表达的病例中显示出潜力。常规生物标志物分析对于优化ATC治疗策略仍至关重要。
肿瘤(癌症)患者之家