Hematopoietic stem cell transplantation for acquired aplastic anemia.
Hematopoietic stem cell transplantation for acquired aplastic anemia.
复制标题
DOI：
10.1097/moh.0000000000000281
复制
发表时间：
2016-11
影响因子：
3.2
通讯作者：
Storb R
中科院分区：
文献类型：
--
作者：
Georges GE;Storb R
同种异体骨髓移植（BMT）治疗严重再生障碍性贫血（SAA）的结果稳步改善，这是由于在调节方案、供体造血细胞来源和支持性护理方面的优化进展。在这里，我们回顾了最近发表的数据，强调了SAA治疗的改进和当前的问题。大约三分之一接受免疫抑制疗法（IST）治疗的AA患者获得了骨髓癌候选基因突变。由于IST最终失败的可能性更大，人类白细胞抗原（HLA）匹配的兄弟姐妹供体BMT是SAA的一线治疗方法。hla匹配非亲属供体（URD） BMT通常推荐给IST失败的患者。然而，在10/10- hla等位基因匹配的URD可以快速识别的年轻患者中，有很强的理由将URD BMT作为一线治疗。移植后使用环磷酰胺（PT-CY）调节方案的hla -单倍体BMT现在是IST失败患者的合理二线治疗。改善的预后导致BMT在SAA治疗中的一线作用增加。hla匹配供体的最佳细胞来源是骨髓。需要进一步的研究来确定hla -单倍体相同供者的最佳调理方案。
There has been steady improvement in outcomes with allogeneic bone marrow transplantation (BMT) for severe aplastic anemia (SAA), due to progress in optimization of the conditioning regimens, donor hematopoietic cell source and supportive care. Here we review recently published data that highlight the improvements and current issues in the treatment of SAA. Approximately one-third of AA patients treated with immune suppression therapy (IST) have acquired mutations in myeloid cancer candidate genes. Because of the greater probability for eventual failure of IST, human leukocyte antigen (HLA)-matched sibling donor BMT is the first-line of treatment for SAA. HLA-matched unrelated donor (URD) BMT is generally recommended for patients who have failed IST. However, in younger patients for whom a 10/10-HLA-allele matched URD can be rapidly identified, there is a strong rationale to proceed with URD BMT as first-line therapy. HLA-haploidentical BMT using post-transplant cyclophosphamide (PT-CY) conditioning regimens, is now a reasonable second-line treatment for patients who failed IST. Improved outcomes have led to an increased first-line role of BMT for treatment of SAA. The optimal cell source from an HLA-matched donor is bone marrow. Additional studies are needed to determine the optimal conditioning regimen for HLA-haploidentical donors.