Complications of transplant for nonmalignant disorders: autoimmune cytopenias, opportunistic infections, and PTLD.

Complications of transplant for nonmalignant disorders: autoimmune cytopenias, opportunistic infections, and PTLD.
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非恶性疾病移植并发症:自身免疫性血细胞减少、机会性感染和 PTLD。

DOI:
10.1016/j.bbmt.2011.10.024
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发表时间:
2012
期刊:
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
影响因子:
--
通讯作者:
Filipovich,Alexandra
Filipovich,Alexandra
中科院分区:
--
文献类型:
--
作者:
Dvorak,ChristopherC;Bollard,CatherineM;El-Bietar,Javier;Filipovich,Alexandra

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在过去的十年中,支持治疗的进步导致了造血细胞移植(HCT)结果的显著改善。尽管患有急性白血病的儿童在非亲属捐献HCT后的1年移植相关死亡率(TRM)接近40%,但在最近的时代,这些比率下降了一半以上,降至约15%。这导致越来越多的替代供体hct被用于各种各样的非恶性疾病,包括原发性免疫缺陷(T细胞和/或吞噬细胞)、血红蛋白病、骨髓衰竭综合征和代谢综合征。典型的白血病患者在接受了数月的免疫抑制化疗后才进入HCT,与之不同的是,许多患有非恶性疾病的患者将在免疫系统完全完好的情况下开始调理方案。这使得这些患者有很高的移植排斥风险,这迫使移植医生采用高度免疫消融的预备方案。即使是进入HCT的免疫系统有缺陷的患者,如患有严重再生障碍性贫血或噬血细胞性淋巴组织细胞增多症的患者,也往往需要显著的免疫消融,因为其潜在倾向于攻击骨髓元素,如移植的造血干细胞。
Advances in supportive care have led to significant improvements in hematopoietic cell transplant (HCT) outcomes over the last decade. Although children with acute leukemia previously had rates of 1-year transplant-related mortality (TRM) following unrelated donor HCT approaching 40%, in a more recent era, these rates have fallen by more than half to approximately 15%[1]. This has led to significantly more alternative donor HCTs being performed for a wide variety of nonmalignant diseases, including primary immunodeficiencies (of T cells and/or phagocytes), hemoglobinopathies, bone marrow failure syndromes, and metabolic syndromes. Unlike a typical patient with leukemia, who enters HCT having received months of immunosuppressive chemotherapy, many patients with a nonmalignant disorder will begin the conditioning regimen with a fully intact immune system. This places those patients at very high risk for graft rejection, which forces transplant physicians to employ preparative regimens that are highly immunoablative. Even patients who enter HCT with defective immune systems, such as those with severe aplastic anemia or hemophagocytic lymphohistiocytosis, tend to require significant immunoablation because of an underlying disposition toward attacking bone marrow elements such as transplanted hematopoietic stem cells.
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