Children with myelodysplastic syndrome (MDS) and increasing mixed chimaerism after allogeneic stem cell transplantation have a poor outcome which can be improved by pre-emptive immunotherapy

Children with myelodysplastic syndrome (MDS) and increasing mixed chimaerism after allogeneic stem cell transplantation have a poor outcome which can be improved by pre-emptive immunotherapy
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DOI:
10.1111/j.1365-2141.2004.05354.x
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发表时间:
2005-03-01
影响因子:
6.5
通讯作者:
Beck, JF
Beck, JF
中科院分区:
医学2区
文献类型:
--
作者:
Bader, P;Niemeyer, C;Beck, JF

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我们最近报道,几乎所有急性白血病和骨髓增生异常综合征(MDS)患儿在同种异体干细胞移植(alloc - sct)后出现混合嵌合(MC)增加的表型都会复发。因此,我们进行了一项针对MDS儿童的前瞻性多中心研究(n = 65;44例晚期MDS患者嵌合性分析显示完全嵌合(CC)/低水平MC/短暂性MC 31例,增高MC 11例,降低MC 2例,对21例伴有难治性细胞减少的MDS患者进行相同分析,发现CC/低水平MC 17例。1例MC增加,3例MC减少。对每个患者进行先发制人的免疫治疗,显示MC增加将无事件生存率从0%提高到50%,如先前研究所见。因此,我们得出结论,先发制人的免疫治疗是一种有效的治疗选择,可以防止儿童MDS患者在同种异体细胞移植后即将复发。
We recently reported that virtually all children with acute leukaemia and myelodysplastic syndrome (MDS) who develop the phenotype of increasing mixed chimaerism (MC) after allogeneic stem cell transplantation (allo-SCT) will relapse. We therefore performed a prospective, multi-centre study focused on children with MDS (n = 65; advanced MDS = 44, refractory cytopenia = 21) after allo-SCT in order to determine to what extent relapse can be prevented by pre-emptive immunotherapy on the basis of increasing MC. Analyses of chimaerism in 44 patients with advanced MDS revealed 31 cases with complete chimaerism (CC)/low-level MC/transient MC, 11 cases with increasing MC and two cases with decreasing MC. The same analyses in 21 MDS patients with refractory cytopenia revealed 17 cases with CC/low-level MC, one case with increasing MC and three cases with decreasing MC. Pre-emptive immunotherapy performed on each patient that showed increasing MC improved event-free survival from 0%, as seen in prior studies, to 50%. We therefore conclude that pre-emptive immunotherapy is an effective treatment option to prevent impending relapse in children with MDS after allo-SCT.