文章：

托泊替康-环磷酰胺方案在成人转移性尤文肉瘤中的疗效与安全性：一项大型、多中心、真实世界研究

Efficacy and Safety of the Topotecan–Cyclophosphamide Regimen in Adult Metastatic Ewing Sarcoma: A Large, Multicenter, Real-World Study

原文发布日期：6 February 2025

DOI: 10.3390/cancers17030550

类型: Article

开放获取: 是

英文摘要：

Background/Objectives: There is an unmet need to improve outcomes in patients with metastatic Ewing sarcoma (ES). This retrospective, multicenter study aimed to evaluate the efficacy and safety of the topotecan–cyclophosphamide (TC) regimen in adult patients with metastatic ES who had previously been treated with chemotherapy. Methods: This study enrolled 75 patients who were treated at five oncology centers in Turkey between 2011 and 2020. Patients were treated with the TC regimen, consisting of topotecan at 0.75 mg/m2/day and cyclophosphamide at 250 mg/m2/day, given daily for 5 days and repeated every 21 days. Results: The median progression-free survival was 3.06 months (95% CI, 2.91–3.22), and the median overall survival was 6.16 months (95% CI, 5.14–7.18). Patients who received the TC regimen in the second line demonstrated longer OS (7.55 months 95% CI, 5.37–14.17) compared to those who received it in the third line or later (5.70 months 95% CI, 4.07–6.60) (p= 0.005). When the TC regimen was used in the second line, the disease control rate was 50%, whereas in the third line or later, the DCR was 10.8%. In the entire group, the DCR was 30.7%. The most common toxicity was transient cytopenia. Conclusions: This study showed that the use of the TC regimen in the second line resulted in better efficacy and overall survival outcomes compared to its use in the third line or later. However, in the entire population, the TC regimen demonstrated only a modest effect on metastatic ES. TC can be considered one of the palliative treatment options for metastatic ES.

摘要翻译： 

背景/目的：目前亟需改善转移性尤文肉瘤（ES）患者的预后。这项回顾性多中心研究旨在评估拓扑替康-环磷酰胺（TC）方案对既往接受过化疗的转移性ES成年患者的疗效和安全性。方法：本研究纳入了2011年至2020年间在土耳其五家肿瘤中心接受治疗的75例患者。患者接受TC方案治疗，具体为拓扑替康0.75 mg/m²/天联合环磷酰胺250 mg/m²/天，每日给药，连续5天，每21天重复一周期。结果：中位无进展生存期为3.06个月（95% CI，2.91–3.22），中位总生存期为6.16个月（95% CI，5.14–7.18）。在二线治疗中接受TC方案的患者总生存期（7.55个月，95% CI，5.37–14.17）较三线及以后接受该方案的患者（5.70个月，95% CI，4.07–6.60）更长（p=0.005）。当TC方案用于二线治疗时，疾病控制率为50%，而用于三线及以后治疗时，疾病控制率为10.8%。在整个研究人群中，疾病控制率为30.7%。最常见的毒性反应为短暂性血细胞减少。结论：本研究表明，与三线及以后使用相比，在二线治疗中使用TC方案能获得更好的疗效和总生存期。然而，在整个研究人群中，TC方案对转移性ES仅显示出有限的疗效。TC方案可被视为转移性ES的姑息治疗选择之一。

原文链接：

Efficacy and Safety of the Topotecan–Cyclophosphamide Regimen in Adult Metastatic Ewing Sarcoma: A Large, Multicenter, Real-World Study