骨髓衰竭综合征和血红蛋白病的移植结果。
Transplant outcomes in bone marrow failure syndromes and hemoglobinopathies.
机构信息
Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, University of Minnesota Medical School, Minneapolis, MN 55455, USA.
出版信息
Semin Hematol. 2010 Jan;47(1):37-45. doi: 10.1053/j.seminhematol.2009.10.004.
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only potential cure for most bone marrow (BM) failure syndromes and hemoglobinopathies. Over the past decade, umbilical cord blood (UCB) has been used more frequently as a stem cell source in patients who lack a suitable BM donor. Although graft failure remains a significant problem, UCB transplantation (UCBT) using the optimal conditioning regimen can be a salvage treatment for patients without a suitable BM donor and warrants evaluation in further prospective studies.
异基因造血干细胞移植（HSCT）是治疗大多数骨髓（BM）衰竭综合征和血红蛋白病的唯一潜在方法。在过去的十年中，由于缺乏合适的 BM 供体，脐带血（UCB）作为干细胞来源在患者中被更频繁地使用。尽管移植物失败仍然是一个重大问题，但使用最佳预处理方案的 UCB 移植（UCBT）可以作为无合适 BM 供体患者的挽救治疗方法，值得在进一步的前瞻性研究中进行评估。
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