Meng Hongfeng, Zhang Boyan, Liu Penghao, Du Yueqi, Zhang Can, Duan Wanru, Chen Zan
Department of Neurosurgery, Xuanwu Hospital, Capital Medical University, 45# Changchun Street, Xicheng District, Beijing, China.
Lab of Spinal Cord Injury and Functional Reconstruction, China International Neuroscience Institute (CHINA-INI), Beijing, China.
Neurosurg Rev. 2025 Jan 21;48(1):70. doi: 10.1007/s10143-025-03204-x.
Chordoma is a rare malignant tumor with a higher incidence in males than in females. There is an increasing number of clinical studies related to tyrosine kinase inhibitors (TKIs), yet the efficacy and safety of different drugs vary. In this single-arm meta-analysis evaluating the efficacy and safety of TKIs for chordoma treatment, 12 studies involving 365 patients were analyzed. The findings suggest that TKIs can improve outcomes, with an objective response rate of 1.7% and 29% based on RECIST and Choi criteria, a median progression-free survival (mPFS) of 8.41 months and a median overall survival (mOS) of 36.6 months. Imatinib, in particular, showed a longer mOS of 39.3 months compared to 25.0 months for other TKIs. However, high toxicity was noted, with a 95% overall incidence of adverse events (AEs), including hypertension, nausea and vomiting, and edema. Serious AEs occurred at a rate of 55%. In subgroup analysis, Imatinib showed a lower incidence of AEs compared to other TKIs. Combination therapy reduced the risk of severe adverse events compared to monotherapy. The study underscores the potential of TKIs to extend survival in chordoma patients but also highlights the need for careful management of treatment-related toxicity. Combining TKIs, especially imatinib, with other treatments may avoid serious adverse events. Further high-quality clinical trials are needed to confirm these findings and optimize treatment protocols.
脊索瘤是一种罕见的恶性肿瘤，男性发病率高于女性。与酪氨酸激酶抑制剂（TKIs）相关的临床研究越来越多，但不同药物的疗效和安全性各不相同。在这项评估TKIs治疗脊索瘤疗效和安全性的单臂荟萃分析中，分析了涉及365例患者的12项研究。结果表明，TKIs可改善预后，根据RECIST和Choi标准，客观缓解率分别为1.7%和29%，中位无进展生存期（mPFS）为8.41个月，中位总生存期（mOS）为36.6个月。特别是伊马替尼，其mOS为39.3个月，比其他TKIs的25.0个月更长。然而，观察到高毒性，不良事件（AEs）的总发生率为95%，包括高血压、恶心呕吐和水肿。严重不良事件的发生率为55%。在亚组分析中，伊马替尼的不良事件发生率低于其他TKIs。与单药治疗相比，联合治疗降低了严重不良事件的风险。该研究强调了TKIs延长脊索瘤患者生存期的潜力，但也突出了谨慎管理治疗相关毒性的必要性。将TKIs，尤其是伊马替尼与其他治疗方法联合使用可能避免严重不良事件。需要进一步的高质量临床试验来证实这些发现并优化治疗方案。
