Department of Haematology II, Azienda Ospedaliera Universitaria San Martino, Genoa, Italy.
Bone Marrow Transplant. 2013 Feb;48(2):198-200. doi: 10.1038/bmt.2012.233. Epub 2012 Nov 26.
Currently at least 75% of patients with severe aplastic anaemia can be successfully transplanted using a matched unrelated donor (UD) haematopoietic SCT (HSCT). For children, outcomes are similar to matched sibling donor (MSD) HSCT. This improvement in outcome over time is likely due to improved HLA tissue typing to identify better matched donors, improvements in the conditioning regimen, particularly fludarabine-based regimens, and improved supportive care. Graft rejection occurs in ∼15% of adults, but is less frequent in children. Chronic GVHD remains a concern but may be reduced by using Alemtuzumab instead of ATG. UD HSCT should be considered early after failure to respond to one course of immunosuppressive therapy, but for children who lack a MSD up front matched UD HSCT may be considered.
目前，至少 75%的重型再生障碍性贫血患者可以通过匹配的无关供体（UD）造血干细胞移植（HSCT）成功移植。对于儿童来说，结果与匹配的同胞供体（MSD）HSCT 相似。随着时间的推移，这种结果的改善可能是由于 HLA 组织分型的改进以确定更好的匹配供体、预处理方案的改进，特别是基于氟达拉滨的方案，以及支持性护理的改进。约 15%的成年人会发生移植物排斥，但在儿童中较少见。慢性移植物抗宿主病仍然是一个问题，但使用阿仑单抗而不是 ATG 可能会减少。在对一线免疫抑制治疗无反应后，应尽早考虑 UD HSCT，但对于缺乏 MSD 的儿童，可能会考虑提前进行匹配的 UD HSCT。
