The Division of Hematology/Oncology, Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto, ON, M5G 1N6, Canada,
Paediatr Drugs. 2013 Dec;15(6):473-92. doi: 10.1007/s40272-013-0037-1.
The Ewing sarcoma family of tumors (ESFT) is defined by cell surface expression of CD99 and a translocation involving EWS and an ETS partner. Cytotoxic chemotherapy remains the benchmark of first- and second-line therapy, and although the majority of patients with localized disease are cured, almost one third of patients relapse or progress from their disease. Moreover, cure remains elusive in most patients who present with distant metastases. In recent years, the ESFT literature has been dominated by reports of attempts at modulating the insulin-like growth factor (IGF) receptor (IGFR). Unfortunately, three phase II studies examining inhibiting antibodies to IGFR-1 published disappointing results. Whether these results were due to failure to modulate the pathway or other limitations in study design and/or patient selection remain unclear. Other novel strategies currently being investigated in ESFT include tyrosine kinase, mammalian target of rapamycin (mTOR), and poly(ADP-ribose) polymerase (PARP) inhibitors.
尤文氏肉瘤家族肿瘤（ESFT）的定义是细胞表面表达 CD99 和涉及 EWS 和 ETS 伙伴的易位。细胞毒性化疗仍然是一线和二线治疗的基准，尽管大多数局限性疾病患者得到治愈，但近三分之一的患者因疾病复发或进展。此外，对于大多数出现远处转移的患者来说，治愈仍然难以实现。近年来，ESFT 文献主要报道了尝试调节胰岛素样生长因子 (IGF) 受体 (IGFR) 的报告。不幸的是，三项研究检查抑制 IGFR-1 抗体的 II 期研究结果令人失望。这些结果是由于未能调节该途径还是由于研究设计和/或患者选择中的其他限制尚不清楚。目前正在 ESFT 中研究的其他新策略包括酪氨酸激酶、雷帕霉素的哺乳动物靶标 (mTOR) 和聚（ADP-核糖）聚合酶 (PARP) 抑制剂。
