Ewing sarcoma is a translocation-associated sarcoma mainly impacting adolescents and young adults. The classic translocation (EWSR1::FLI1) leads to a fusion oncoprotein that functions as an aberrant transcription factor. As such, the oncogenic driver of this disease has been difficult to target pharmacologically and, therefore, the systemic therapies used to treat patients with Ewing sarcoma have typically been non-selective cytotoxic chemotherapy agents. The current review highlights recent clinical trials from the last decade that provide the evidence base for contemporary drug therapy for patients with Ewing sarcoma, while also highlighting novel therapies under active clinical investigation in this disease. We review recent trials that have led to the establishment of interval-compressed chemotherapy as an international standard for patients with newly diagnosed localized disease. We further highlight recent trials that have shown a lack of demonstrable benefit from high-dose chemotherapy or IGF-1R inhibition for patients with newly diagnosed metastatic disease. Finally, we provide an overview of chemotherapy regimens and targeted therapies used in the management of patients with recurrent Ewing sarcoma.

尤文肉瘤是一种易位相关肉瘤，主要影响青少年和年轻人。经典易位（EWSR1::FLI1）导致融合癌蛋白发挥异常转录因子的作用。因此，这种疾病的致癌驱动因素很难在药理学上靶向，因此，用于治疗尤文肉瘤患者的全身疗法通常是非选择性细胞毒性化疗药物。当前的综述重点介绍了过去十年的最新临床试验，这些试验为尤文肉瘤患者的当代药物治疗提供了证据基础，同时也重点介绍了该疾病正在积极临床研究中的新疗法。我们回顾了最近的试验，这些试验导致间隔压缩化疗成为新诊断的局部疾病患者的国际标准。我们进一步强调最近的试验表明，高剂量化疗或 IGF-1R 抑制对于新诊断的转移性疾病患者缺乏明显的益处。最后，我们概述了用于治疗复发性尤文肉瘤患者的化疗方案和靶向治疗。