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脐带血移植的最新进展。

Update on umbilical cord blood transplantation.

作者信息

Kurtzberg Joanne

机构信息

The Pediatric Blood and Marrow Transplant Program, The Carolinas Cord Blood Bank, Duke University Medical Center, Durham, North Carolina 27705, USA.

出版信息

Curr Opin Pediatr. 2009 Feb;21(1):22-9. doi: 10.1097/mop.0b013e32832130bc.

DOI:10.1097/mop.0b013e32832130bc
PMID:19253461
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3682484/
Abstract

PURPOSE OF REVIEW

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.

RECENT FINDINGS

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.

SUMMARY

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.

摘要

综述目的

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。

最新发现

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。

总结

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。

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本文引用的文献

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Results of the Cord Blood Transplantation Study (COBLT): clinical outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with hematologic malignancies.脐血移植研究(COBLT)结果:血液系统恶性肿瘤儿科患者接受无关供者脐血移植的临床结局
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Emerging trends in transplantation of inherited metabolic diseases.
脐血有核细胞的单细胞转录组图谱。
Gigascience. 2019 May 1;8(5). doi: 10.1093/gigascience/giz047.
4
Diversity of dendritic cells generated from umbilical cord or adult peripheral blood precursors.源自脐带或成人外周血前体细胞的树突状细胞的多样性。
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Sickle Cell Disease: Advances in Treatment.镰状细胞病:治疗进展
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Proliferation extent of CD34 cells as a key parameter to maximize megakaryocytic differentiation of umbilical cord blood-derived hematopoietic stem/progenitor cells in a two-stage culture protocol.在两阶段培养方案中,CD34细胞的增殖程度作为一个关键参数,可使脐带血来源的造血干细胞/祖细胞的巨核细胞分化最大化。
Biotechnol Rep (Amst). 2014 Jul 16;4:50-55. doi: 10.1016/j.btre.2014.07.002. eCollection 2014 Dec.
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Stem Cell Banking for Regenerative and Personalized Medicine.用于再生医学和个性化医疗的干细胞库
Biomedicines. 2014 Feb 26;2(1):50-79. doi: 10.3390/biomedicines2010050.
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Cellular transplant therapies for globoid cell leukodystrophy: Preclinical and clinical observations.球状细胞脑白质营养不良的细胞移植疗法:临床前和临床观察
J Neurosci Res. 2016 Nov;94(11):1180-8. doi: 10.1002/jnr.23782.
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Factors That Influence a Mother's Willingness to Preserve Umbilical Cord Blood: A Survey of 5120 Chinese Mothers.影响母亲保存脐带血意愿的因素:对5120名中国母亲的调查
PLoS One. 2015 Dec 9;10(12):e0144001. doi: 10.1371/journal.pone.0144001. eCollection 2015.
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Correction of chronic granulomatous disease after second unrelated-donor umbilical cord blood transplantation.第二次非亲属供者脐带血移植后慢性肉芽肿病的纠正
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Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: a comparison study.无关供者脐血与骨髓移植治疗儿童急性白血病的疗效:一项比较研究。
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Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure.欧洲黏多糖贮积症Ⅰ型造血干细胞移植的结局:移植物失败的危险因素分析
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Busulfan/melphalan/antithymocyte globulin followed by unrelated donor cord blood transplantation for treatment of infant leukemia and leukemia in young children: the Cord Blood Transplantation study (COBLT) experience.白消安/美法仑/抗胸腺细胞球蛋白联合非血缘供者脐血移植治疗婴儿白血病和幼儿白血病:脐血移植研究(COBLT)经验
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Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency.脐带干细胞移植治疗原发性免疫缺陷的单中心经验
Bone Marrow Transplant. 2005 Aug;36(4):295-9. doi: 10.1038/sj.bmt.1705054.