Department of Pediatrics, University of Minnesota Pediatric Blood and Marrow Transplant Program, Minneapolis, MN, USA.
Semin Hematol. 2010 Jan;47(1):79-85. doi: 10.1053/j.seminhematol.2009.10.001.
Primary immune deficiencies (PIDs) are rare diseases, and most are lethal without appropriate intervention. Hematopoietic cell transplantation (HCT) can cure the majority of patients, but most lack a suitable matched related donor. Alternative donor stem cells (mismatched related donor bone marrow, unrelated donor bone marrow, and unrelated donor umbilical cord blood [UCB]) are therefore frequently required. Published data comparing outcomes after alternative donor transplant for PID are scarce. The outcomes and potential advantage and disadvantages of each alternative stem cell source are discussed in this chapter. Although there are insufficient prospective data to make meaningful comparisons between the alternative stem cell sources, the results presented here demonstrate clearly that the use of UCB transplantation for PID is a viable option and may be advantageous in many situations.
原发性免疫缺陷病（PID）较为罕见，大多数疾病如不及时干预则危及生命。造血干细胞移植（HCT）可治愈大多数患者，但大多数患者缺乏合适的配型相关供体。因此，经常需要替代供体干细胞（不合型相关供体骨髓、无关供体骨髓和无关供体脐带血[UCB]）。目前，关于替代供体移植治疗 PID 的结果比较数据较少。本章讨论了每种替代干细胞来源的结果以及潜在的优势和劣势。尽管替代干细胞来源之间的前瞻性数据不足，无法进行有意义的比较，但这里呈现的结果清楚地表明，使用 UCB 移植治疗 PID 是一种可行的选择，在许多情况下可能具有优势。
