如何在获得性和遗传性骨髓衰竭综合症患者中,优化HLA匹配相关的造血干细胞移植的结果。
How to optimize outcome of patients undergoing HLA-matched related haematopoietic stem cell transplantation in acquired and inherited bone marrow failure syndromes.
发表日期:2023 Aug 16
作者:
Stefano Giardino, Filomena Pierri, Maura Faraci
来源:
BRITISH JOURNAL OF HAEMATOLOGY
摘要:
在具有健康配型相关供者(MRD)的情况下,经过弱化程度的预处理方案进行前期同种异体造血干细胞移植(allo-HSCT)是儿童获得性重型再障碍性贫血(aSAA)和先天性骨髓功能衰竭综合症(iBMFs)的标准治疗方法。Alsultan等人的论文报道了低剂量环磷酰胺、氟达拉滨和胸腺球蛋白预处理的MRD HSCT的安全性和疗效,在aSAA和非Fanconi iBMFs方面加强了免疫抑制方法在allo-HSCT中针对特定非恶性疾病亚群的关键作用的概念,为讨论在BMF患者中实现MRD HSCT后患者长期生存的关键要点提供了机会。评述文章:Alsultan等人。使用低剂量环磷酰胺、氟达拉滨和胸腺球蛋白进行人类白细胞抗原配型相关造血干细胞移植治疗儿童重型再障碍性贫血。《英国血液学杂志》2023年(在线发表前)。doi:10.1111/bjh.19004。© 2023 The Authors. British Journal of Haematology,British Society for Haematology和John Wiley&Sons Ltd.发表。
Up-front allogeneic haematopoietic stem cell transplantation (allo-HSCT) after a reduced intensity conditioning regimen is the standard treatment in children with acquired severe aplastic anaemia (aSAA) and inherited bone marrow failure syndromes (iBMFs) in the presence of a healthy matched related donor (MRD). The paper by Alsultan et al. report the safety and efficacy of MRD HSCT conditioned with low-dose cyclophosphamide, fludarabine and thymoglobulin in both aSAA and non-Fanconi iBMFs, strengthening the concept of the pivotal role of immunosuppressive approach in allo-HSCT for specific subgroups of non-malignant diseases requiring a reduced risk of toxicities, offering the opportunity to discuss the essential points for achieving patients' long-term survival after MRD HSCT in BMF. Commentary on: Alsultan et al. Human leucocyte antigen-matched related haematopoietic stem cell transplantation using low-dose cyclophosphamide, fludarabine and thymoglobulin in children with severe aplastic anaemia. Br J Haematol 2023 (Online ahead of print). doi: 10.1111/bjh.19004.© 2023 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd.