Department of Paediatrics, Aarhus University Hospital Skejby, Aarhus, Denmark.
Br J Haematol. 2014 Jul;166(1):23-33. doi: 10.1111/bjh.12900. Epub 2014 Apr 22.
The last decades have seen parallel improvements in chemotherapy-based and haematopoietic stem cell transplantation (HSCT) regimens for acute myeloid leukaemia (AML) in children. There has been no consensus on indication for HSCT. Reserving HSCT for high-risk and relapsed patients spare many patients from the long-term toxicity of this treatment. The results of matched unrelated donor HSCT equal family donor transplantation and the presence of a matched sibling should no longer be a transplant indication. Minimal residual disease measured by flow cytometry may identify poor responders benefitting from HSCT in first complete remission (CR1) and those with a favourable response to induction therapy who do not need HSCT even with adverse cytogenetic aberrations. FLT3-internal tandem duplication without NPM1 mutation has a very high relapse rate despite favourable response and HSCT is indicated in CR1 in these cases. Finding the optimal indications for HSCT is a delicate balance between risk of relapse and late effects.
过去几十年中，儿童急性髓系白血病（AML）的化疗方案和造血干细胞移植（HSCT）方案都取得了进展。然而，HSCT 的适应证尚未达成共识。将 HSCT 保留给高危和复发患者，可以使许多患者免受这种治疗的长期毒性影响。匹配的无关供体 HSCT 的结果与家族供体移植相当，并且存在匹配的同胞供体不应再成为移植的适应证。通过流式细胞术测量微小残留病可以识别出在首次完全缓解（CR1）中受益于 HSCT 的不良反应者，以及那些对诱导治疗有良好反应但即使存在不良细胞遗传学异常也不需要 HSCT 的患者。FLT3 内部串联重复而无 NPM1 突变尽管反应良好，但复发率很高，在这种情况下，CR1 时应进行 HSCT。找到 HSCT 的最佳适应证是在复发风险和晚期效应之间取得微妙平衡。
