Department of Pediatric Hematology, Immunology and Oncology, APHM, La Timone Children's Hospital, Marseille, France.
REMAP4KIDS, CNRS, INSERM, CRCM, Aix Marseille University, Marseille, France.
Pediatr Blood Cancer. 2024 Aug;71(8):e31076. doi: 10.1002/pbc.31076. Epub 2024 May 22.
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.
节拍化疗为基础的联合治疗方案在复发性/难治性恶性肿瘤中受到关注。临床前和临床研究表明，节拍依托泊苷和阿昔替尼具有活性。我们报告了 6 例儿童脑肿瘤患者采用阿昔替尼和节拍依托泊苷治疗的回顾性经验，这是一种“超适应证”的联合治疗。3 例髓母细胞瘤患者出现部分缓解；1 例间变性横纹肌样瘤（ATRT）患者疾病持续稳定（12 个月）；2 例髓母细胞瘤患者疾病进展。2 例患者出现 3-4 级毒性（血小板减少、贫血、腹泻、乏力）。阿昔替尼-依托泊苷联合方案在复发性/难治性脑肿瘤的大量预处理患者中显示出疗效信号。这些结果是基于真实世界的观察，需要在 I/II 期试验中进行正式评估。
