Cousson Sophie, Calvo Charlotte, Goldwirt Lauriane, Simonin Mathieu, Roupret-Serzec Julie, Dourthe Marie Émilie, Strullu Marion, Baruchel André, Dalle Jean-Hugues, Brethon Benoît
Pediatric Hematology and Immunology Department, Robert Debré Academic Hospital, GHU AP-HP Nord Université Paris Cité, Paris, France.
Pharmacology Department, Saint-Louis Academic Hospital, GHU AP-HP Nord Université Paris Cité, Paris, France.
Br J Haematol. 2025 Jan;206(1):209-214. doi: 10.1111/bjh.19849. Epub 2024 Oct 24.
BCL-2 inhibitor venetoclax demonstrates promising efficacy in paediatric relapsed/refractory acute myeloid leukaemia (r/r AML). This retrospective analysis evaluated 12 patients treated with venetoclax-based regimens under compassionate use for r/r myeloid malignancies. The overall response rate (ORR) was 41.6%, with complete response (CR) achieved in 33% of patients. Three patients successfully underwent allogeneic haematopoietic scell transplantation (HSCT) after venetoclax bridging therapy. Venetoclax demonstrated a favourable safety profile with manageable side effects. These findings suggest venetoclax's potential as a valuable therapeutic option for paediatric r/r AML, particularly for heavily pretreated patients. Further investigation in larger multicentre trials is warranted to refine treatment strategy.
BCL-2抑制剂维奈克拉在儿童复发/难治性急性髓系白血病（r/r AML）中显示出有前景的疗效。这项回顾性分析评估了12例因r/r髓系恶性肿瘤在同情用药情况下接受基于维奈克拉方案治疗的患者。总缓解率（ORR）为41.6%，33%的患者实现了完全缓解（CR）。3例患者在维奈克拉桥接治疗后成功接受了异基因造血干细胞移植（HSCT）。维奈克拉显示出良好的安全性，副作用可控。这些发现表明维奈克拉作为儿童r/r AML，特别是重度预处理患者的一种有价值的治疗选择具有潜力。有必要在更大规模的多中心试验中进行进一步研究以完善治疗策略。
