氟达拉滨、Campath和低剂量环磷酰胺(fclow)伴或不伴TBI调节可使重度再生障碍性贫血儿童移植预后良好

IF 4.3 Q1 Medicine
Ravi M. Shah , Tony H. Truong , Michel T. Leaker , Nicola A.M. Wright , Doan Le , MacGregor Steele , Aisha A.K. Bruce , Sunil Desai , Gregory M.T. Guilcher , Victor Lewis
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引用次数: 4

摘要

各种降低强度调理方案用于特发性严重再生障碍性贫血(SAA)患者的异基因造血细胞移植(HSCT)。我们描述了15名接受相关或非相关供体移植的儿童使用氟达拉滨、Campath和低剂量环磷酰胺(fclow)调节。非亲属供体HSCT增加2gy的全身照射(TBI)。中位随访时间为2.3年,无失败生存率为100%,感染和毒性发生率低。未发生III至IV级急性移植物抗宿主病(GVHD)。所有患者在造血干细胞移植后都有完全的供体骨髓嵌合,甚至在T细胞谱系中有混合嵌合。慢性GVHD的缺失和长期稳定的混合供体T细胞嵌合证实了儿童SAA在FCClow(±TBI)条件移植后的免疫耐受。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Fludarabine, Campath, and Low-Dose Cyclophosphamide (FCClow) with or without TBI Conditioning Results in Excellent Transplant Outcomes in Children with Severe Aplastic Anemia

Various reduced-intensity conditioning regimens are in use for allogeneic hematopoietic cell transplant (HSCT) in patients with idiopathic severe aplastic anemia (SAA). We describe the use of fludarabine, Campath, and low-dose cyclophosphamide (FCClow) conditioning in 15 children undergoing related or unrelated donor transplants. Total body irradiation (TBI) of 2 Gy was added for unrelated donor HSCT. At a median follow-up of 2.3 years, the failure-free survival was 100%, with low rates of infection and toxicity. There was no occurrence of grade III to IV acute graft-versus-host disease (GVHD). All patients had full donor myeloid chimerism post-HSCT, even with mixed chimerism in the T cell lineage. The absence of chronic GVHD and long-term stable mixed donor T cell chimerism confirms immune tolerance following FCClow (± TBI) conditioned transplantation in children with SAA.

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来源期刊
CiteScore
6.60
自引率
0.00%
发文量
1061
审稿时长
3-6 weeks
期刊介绍: Biology of Blood and Marrow Transplantation publishes original research reports, reviews, editorials, commentaries, letters to the editor, and hypotheses and is the official publication of the American Society for Transplantation and Cellular Therapy. The journal focuses on current technology and knowledge in the interdisciplinary field of hematopoetic stem cell transplantation.
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