Wang Yitian, Min Li, Zhou Yong, Luo Yi, Duan Hong, Tu Chongqi
Department of Orthopedics, West China Hospital, Sichuan University, Chengdu, Sichuan 610041, People's Republic of China,
Cancer Manag Res. 2018 Dec 11;10:6835-6842. doi: 10.2147/CMAR.S181087. eCollection 2018.
Ewing's sarcoma (ES) is a highly aggressive and metastatic neoplasm occurring mainly in children and young adults. The standard treatment of localized ES requires a combination of surgery, chemotherapy, and radiotherapy. Although the 5-year survival rate for local ES has improved, the survival rate and prognosis are still very poor for metastatic or recurrent ES patients. The aim of this study was to investigate the efficacy and safety of apatinib, a specific vascular endothelial growth factor receptor 2 inhibitor, in ES patients.
This retrospective analysis involved eleven patients with ES not amenable to curative treatment. All patients suffered poor responses to two cycles of chemotherapy (vincristine, doxorubicin, and cyclophosphamide). Apatinib 500 mg (or 250 mg) was given daily. Tumor responses were assessed according to the Response Evaluation Criteria in Solid Tumors 1.1. Survival analysis was performed by the Kaplan-Meier test. The safety profile was also recorded.
The mean age of the patients was 18 (range, 10-31) years. The 12-month overall survival and progression-free survival rates were 90% and 72%, respectively. Four patients achieved partial response, and four patients achieved stable disease, with objective response rate of 40%. The median follow-up in our study was 16 months (range, 3-26 months). The most common adverse events included hand-foot skin reaction (n=5; 45%), oral ulcers (n=4; 36%), and gastrointestinal discomfort (n=4; 36%).
Apatinib may provide as second- or first-line treatment options for ES patients, particularly in chemoresistant cases. Further studies with more cases and longer follow-up will be necessary to determine the clinical efficacy and safety of apatinib in ES patients.
尤因肉瘤（ES）是一种主要发生于儿童和青年的高侵袭性转移性肿瘤。局限性ES的标准治疗需要手术、化疗和放疗联合应用。尽管局限性ES的5年生存率有所提高，但转移性或复发性ES患者的生存率和预后仍然很差。本研究的目的是探讨特异性血管内皮生长因子受体2抑制剂阿帕替尼在ES患者中的疗效和安全性。
本回顾性分析纳入了11例无法进行根治性治疗的ES患者。所有患者对两周期化疗（长春新碱、阿霉素和环磷酰胺）反应不佳。每日给予阿帕替尼500mg（或250mg）。根据实体瘤疗效评价标准1.1评估肿瘤反应。采用Kaplan-Meier检验进行生存分析。同时记录安全性情况。
患者的平均年龄为18岁（范围10 - 31岁）。12个月的总生存率和无进展生存率分别为90%和72%。4例患者达到部分缓解，4例患者病情稳定，客观缓解率为40%。本研究的中位随访时间为16个月（范围3 - 26个月）。最常见的不良事件包括手足皮肤反应（n = 5；45%）、口腔溃疡（n = 4；36%）和胃肠道不适（n = 4；36%）。
阿帕替尼可为ES患者提供二线或一线治疗选择，尤其是对化疗耐药的病例。需要进一步开展更多病例和更长随访时间的研究，以确定阿帕替尼在ES患者中的临床疗效和安全性。
