Kurtzberg Joanne
The Pediatric Blood and Marrow Transplant Program, The Carolinas Cord Blood Bank, Duke University Medical Center, Durham, North Carolina 27705, USA.
Curr Opin Pediatr. 2009 Feb;21(1):22-9. doi: 10.1097/mop.0b013e32832130bc.
2008 marks the 20th anniversary of the first use of umbilical cord blood (UCB) as a source of donor cells for hematopoietic stem cell transplantation. In those early days, there was great doubt and skepticism about the utility of UCB as a source of hematopoietic stem cells. Doubts about whether UCB, containing 10-20x fewer cells than bone marrow, had sufficient cells to durably engraft a myeloablated patient and, after demonstration that engraftment occurred with less graft-versus-host disease, whether it would confer graft-versus-leukemia activity were raised.
Transplantation with UCB is effective in the treatment of children with hematological malignancies, marrow failure, immunodeficiencies, hemoglobinopathies and inherited metabolic diseases. Transplantation without full human leukocyte antigen matching is possible and, despite a lower incidence of graft-versus-host disease, graft-versus-leukemia is preserved. The number of cells in a single UCB can be limiting, but the use of two UCBs for a single transplant shows promise to overcome this obstacle.
Cord blood transplantation is now an established field with enormous potential. UCB increases access to transplantation therapy for many patients unable to identify a fully matched adult donor. In the future, it may emerge as a source of cells for cellular therapies focused on tissue repair and regeneration.
2008年是首次将脐带血(UCB)用作造血干细胞移植供体细胞来源的20周年。在早期,人们对UCB作为造血干细胞来源的效用存在很大疑问和怀疑。有人质疑,UCB所含细胞数量比骨髓少10至20倍,是否有足够的细胞使接受大剂量化疗的患者持久植入,以及在证明植入后移植物抗宿主病较少发生后,它是否会具有移植物抗白血病活性。
UCB移植在治疗患有血液系统恶性肿瘤、骨髓衰竭、免疫缺陷、血红蛋白病和遗传性代谢疾病的儿童方面是有效的。在没有完全人类白细胞抗原匹配的情况下进行移植是可行的,并且尽管移植物抗宿主病的发生率较低,但移植物抗白血病作用得以保留。单个UCB中的细胞数量可能有限,但单次移植使用两份UCB有望克服这一障碍。
脐带血移植现在是一个已确立的领域,具有巨大潜力。UCB增加了许多无法找到完全匹配的成年供体的患者获得移植治疗的机会。未来,它可能会成为专注于组织修复和再生的细胞治疗的细胞来源。