阿帕替尼治疗软组织肉瘤的有效性及安全性27例回顾分析  

Efficacy and safety of apatinib in treating soft tissue sarcoma: a retrospective analysis of twenty-seven cases

作　　者：龚涛军 何梅[2] 王一天 闵理[1] 周勇[1] 罗翼[1] 段宏[1] 屠重棋[1] GONG Tao-jun;HE Mei;WANG Yi-tian;MIN Li;ZHOU Yong;LUO Yi;DUAN Hong;TU Chong-qi(Department of Orthopedics,West China Hospital,Sichuan University,Chengdu,Sichuan,610041,China)

机构地区：[1]四川大学华西医院骨科,成都610041 [2]四川大学华西医院手术室,成都610041

出　　处：《中国骨与关节杂志》2020年第5期341-347,共7页Chinese Journal of Bone and Joint

基　　金：国家自然科学基金(81801852);成都科学与技术项目基金(2017-CY02-00032-GX)。

摘　　要：目的探究特异性血管内皮生长因子受体-2 抑制剂阿帕替尼治疗软组织肉瘤、滑膜肉瘤(synovial sarcoma,SS) 及转移性腺泡样肉瘤 (alveolar soft part sarcoma,ASPS) 的有效性及安全性。方法本研究纳入 27 例,平均年龄 30.2 (8~71) 岁,男 15 例,女 12 例,其中 21 例诊断为 SS,6 例诊断为转移性 ASPS。患者每日口服 1 次阿帕替尼 500 mg (或年龄<10 岁时 250 mg),根据实体瘤治疗评价指南 (RECIST 1.1)评估肿瘤反应性,Kaplan-Meier 试验用于生存分析,同时记录用药不良反应。结果平均随访 13.7 (2.8^-34.1) 个月。患者中位无进展生存期 (PFS) 为 12.38 个月 (95% CI,12.23-NE)。但是中位总体生存期 (OS)尚未获得。1 例 ASPS 达到了完全缓解 (CP),14 例达到了部分缓解 (PR),8 例为稳定状态 (SD),4 例 SS 出现疾病进展 (PD),客观缓解率 (ORR) 为 55.6% (12.16~100.00),疾病控制率 (DCR) 为 85.2% (34.39~100.00);常见的不良反应包括手足皮肤反应 [13 / 27 (48.1%)],胃肠道反应 [13 / 27 (48.1%)],毛发色素减退 [8 / 17 (29.6%)],口腔溃疡 [6 / 27 (22.2%)]。结论对于软组织肉瘤患者而言,阿帕替尼可作为三线或二线甚至一线治疗选择用药,其有效性和安全性仍须多中心、大样本、前瞻性研究的长期随访结果验证。Objective To investigate the efficacy and safety of specific vascular endothelial growth factor receptor-2 inhibitor apatinib in soft tissue sarcoma: synovial sarcoma(SS) and alveolar soft part sarcoma(ASPS). Methods This study involved 27 patients(15 males, 12 females) with a mean age of 30.2 years(range: 8-71 years). Twenty-one patients were diagnosed as SS, while 6 as ASPS. Apatinib was administered at the dose of 500 mg once per day(or 250 mg when age < 10). Tumor responses were assessed according to the Response Evaluation Criteria in Solid Tumors(RECIST 1.1) guidelines. Survival analysis was performed using the Kaplan–Meier test, and a safety profile was recorded. Results The mean follow-up was 13.7 months(range: 2.8-34.1 months). The median progression-free survival(PFS) was 12.38 months(95% CI,12.23-NE). The Median overall survival(OS) had not been reached yet. One ASPS patient was assessed as complete response(CR), 14 patients as partial response(PR), 8 patients as stable disease(SD). Four SS patients were assessed as progressive disease(PD). The Objective Response Rate(ORR) was 55.6%(12.16-100.00). The Disease Control Rate(DCR) was 85.2%(34.39-100.00). The most common adverse events(AEs) included hand–foot skin reaction [ 13/27(48.1%) ], gastrointestinal discomfort [ 13/27(48.1%) ], hair hypopigmentation [ 8/17(29.6%) ], and oral ulcers [ 6/27(22.2%) ]. Conclusions This study provides the evidence of the efficacy and safety of apatinib as a third or second-line even first-line medical option for patients with soft tissue sarcoma. However, prospective multi-center research with large samples and long-term followup is needed.

关 键 词：软组织肉瘤 滑膜肉瘤 腺泡样肉瘤 阿帕替尼 血管内皮生长因子 

分 类 号：R738.6[医药卫生—肿瘤]