阿昔替尼是一种口服的血管内皮生长因子受体抑制剂，对肾癌、甲状腺癌和胰腺癌具有抗肿瘤活性。Axi-STS是一项病理分层、非随机、开放标签的多中心II期试验，研究连续应用阿昔替尼治疗≥16岁、临床表现≤2级、病理确诊的晚期/转移性软组织肉瘤（STS）患者。患者按照四个肿瘤亚型分组招募，分别进行分析：血管肉瘤，平滑肌肉瘤，滑膜肉瘤或其他符合条件的STS。主要结局指标是12周进展生存率（PFS12）。根据PFS12为40％的活性定义，使用Simon的二阶段设计确定每个分组的样本量为33名患者。在2010年8月31日至2016年1月29日期间，共招募了145名患者：38名血管肉瘤，37名平滑肌肉瘤，36名滑膜肉瘤和34名其他亚型。每个分组的PFS12率分别为42％（95％下限置信区间（LCI）；29），45％（95％ LCI；32），57％（95％ LCI；42）和33％（95％ LCI；21）。共有74个严重不良事件，其中两例与治疗相关的死亡事件分别为肺出血和胃肠道出血。疲劳和高血压是最常见的3级不良事件。阿昔替尼在所有研究的STS亚型中均显示出临床活性。不良事件概况可接受，支持进一步在III期试验中进行研究。ISRCTN 60791336.©2023.作者

Axitinib is an oral vascular endothelial growth factor receptor inhibitor with anti-tumour activity in renal, thyroid, and pancreatic cancer.Axi-STS was a pathologically-stratified, non-randomised, open-label, multi-centre, phase II trial of continuous axitinib treatment in patients ≥16 years, performance status ≤2, with pathologically-confirmed advanced/metastatic soft tissue sarcoma (STS). Patients were recruited within four tumour strata, each analysed separately: angiosarcoma, leiomyosarcoma, synovial sarcoma, or other eligible STSs. The primary outcome was progression-free survival at 12 weeks (PFS12). A Simon's two-stage design with activity defined as PFS12 rate of 40% determined a sample size of 33 patients per strata.Between 31-August-2010 and 29-January-2016, 145 patients were recruited: 38 angiosarcoma, 37 leiomyosarcoma, 36 synovial sarcoma, and 34 other subtypes. PFS12 rate for each stratum analysed was 42% (95% lower confidence interval (LCI); 29), 45% (95% LCI; 32), 57% (95% LCI; 42), and 33% (95% LCI; 21), respectively. There were 74 serious adverse events including two treatment-related deaths of pulmonary haemorrhage and gastrointestinal bleeding. Fatigue and hypertension were the most common grade 3 adverse events.Axitinib showed clinical activity in all STS strata investigated. The adverse event profile was acceptable, supporting further investigation in phase III trials.ISRCTN 60791336.© 2023. The Author(s).