Abstract：
Metronomic chemotherapy-based combinations have received interest for relapsed/refractory malignancies. Preclinical and clinical studies showed activity of metronomic etoposide and axitinib. We report our retrospective experience in six children treated with axitinib and metronomic etoposide for refractory/relapsed brain tumors as an \"off-label\" combination. Three patients with medulloblastoma experienced partial response; one patient with atypical teratoid rhabdoid tumor (ATRT) displays an ongoing stable disease (12 months); two patients with medulloblastoma had progressive disease. Grade 3-4 toxicities were observed in two patients (thrombocytopenia, anemia, diarrhea, fatigue). The axitinib-etoposide combination shows signals of efficacy in heavily pretreated patients with relapsed/refractory brain tumors. These results were based on real-world observation and will need formal evaluation in a phase I/II trial.
摘要：
基于节制化疗的组合已引起复发/难治性恶性肿瘤的兴趣。临床前和临床研究显示节拍依托泊苷和阿西替尼的活性。我们报告了我们在6例接受阿西替尼和节拍依托泊苷治疗难治性/复发性脑肿瘤的“标签外”组合的儿童的回顾性经验。三名髓母细胞瘤患者出现部分反应；一名非典型畸胎瘤样横纹肌样瘤（ATRT）患者表现出持续稳定的疾病（12个月）；两名髓母细胞瘤患者患有进行性疾病。在两名患者中观察到3-4级毒性(血小板减少症，贫血,腹泻,疲劳)。阿昔替尼-依托泊苷组合在严重预处理的复发/难治性脑肿瘤患者中显示出疗效信号。这些结果是基于现实世界的观察，需要在I/II期试验中进行正式评估。
