Winnicki Camille, Leblond Pierre, Bourdeaut Franck, Pagnier Anne, Paluenzela Gilles, Chastagner Pascal, Duhil-De Benaze Gwenaelle, Min Victoria, Sudour-Bonnange Hélène, Piette Catherine, Entz-Werle Natacha, Chabaud Sylvie, André Nicolas
Department of Pediatric Immunology, Hematology and Oncology, Children Hospital of La Timone, Assistance Publique Hôpitaux de Marseille, 13005 Marseille, France.
Department of Pediatric Oncology, Institut d'Hématologie et d'Oncologie Pédiatrique, Centre Léon Bérard, 69008 Lyon, France.
J Clin Med. 2023 Feb 10;12(4):1415. doi: 10.3390/jcm12041415.
Relapses in pediatric high-risk brain tumors remain unmet medical needs. Over the last 15 years, metronomic chemotherapy has gradually emerged as an alternative therapeutic approach.
This is a national retrospective study of patients with relapsing pediatric brain tumors treated according to the MEMMAT or MEMMAT-like regimen from 2010 to 2022. Treatment consisted of daily oral thalidomide, fenofibrate, and celecoxib, and alternating 21-day cycles of metronomic etoposide and cyclophosphamide associated with bevacizumab and intraventricular chemotherapy.
Forty-one patients were included. The most frequent malignancies were medulloblastoma (22) and ATRT (8). Overall, the best responses were CR in eight patients (20%), PR in three patients (7%), and SD in three patients (7%), for a clinical benefit rate of 34%. The median overall survival was 26 months (IC95% = 12.4-42.7), and median EFS was 9.7 months (IC95% = 6.0-18.6). The most frequent grade ¾ toxicities were hematological. Dose had to be adjusted in 27% of the cases. There was no statistical difference in outcome between full or modified MEMMAT. The best setting seems to be when MEMMAT is used as a maintenance and at first relapse.
The metronomic MEMMAT combination can lead to sustained control of relapsed high-risk pediatric brain tumors.
小儿高危脑肿瘤的复发仍是未满足的医疗需求。在过去15年中，节拍化疗已逐渐成为一种替代治疗方法。
这是一项全国性回顾性研究，研究对象为2010年至2022年期间根据MEMMAT或类似MEMMAT方案治疗的复发性小儿脑肿瘤患者。治疗包括每日口服沙利度胺、非诺贝特和塞来昔布，以及与贝伐单抗和脑室内化疗联合的节拍依托泊苷和环磷酰胺的21天交替周期。
纳入41例患者。最常见的恶性肿瘤是髓母细胞瘤（22例）和非典型畸胎样/横纹肌样瘤（ATRT，8例）。总体而言，最佳反应为8例患者完全缓解（CR，20%），3例患者部分缓解（PR，7%），3例患者疾病稳定（SD，7%），临床获益率为34%。中位总生存期为26个月（95%置信区间[IC95%]=12.4 - 42.7），中位无进展生存期为9.7个月（IC95% = 6.0 - 18.6）。最常见的3/4级毒性为血液学毒性。27%的病例需要调整剂量。完整或改良的MEMMAT方案在疗效上无统计学差异。最佳的应用场景似乎是在MEMMAT用作维持治疗以及首次复发时。
节拍性MEMMAT联合方案可导致复发性高危小儿脑肿瘤得到持续控制。
