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采用低毒性方案成功进行无关供者脐血移植治疗重型β-地中海贫血3级患者。

Successful unrelated umbilical cord blood transplantation for class 3 β-thalassemia major using a reduced-toxicity regimen.

作者信息

Soni Sandeep, Breslin Nancy, Cheerva Alexandra

机构信息

Division of Heme/Onc/BMT, Nationwide Children's Hospital, The Ohio State University, Columbus, OH, USA.

出版信息

Pediatr Transplant. 2014 Mar;18(2):E41-3. doi: 10.1111/petr.12201. Epub 2013 Dec 16.

DOI:10.1111/petr.12201
PMID:24330091
Abstract

Unrelated umbilical CB transplant for class 3 β-thalassemia major is associated with an increased risk of mortality and non-engraftment. We describe two patients who underwent successful unrelated umbilical CB transplant using a novel reduced-toxicity preparative regimen. This regimen may be sufficiently immunosuppressive and myeloablative to ensure engraftment with reduced risks of toxicity and mortality. Close monitoring of HHV-6 viral load is advised for patients undergoing transplant with this regimen.

摘要

无关供者脐血移植治疗重型β地中海贫血3级与死亡和未植入风险增加相关。我们描述了两名患者,他们使用一种新型低毒性预处理方案成功接受了无关供者脐血移植。该方案可能具有足够的免疫抑制和清髓作用,以确保植入,同时降低毒性和死亡风险。建议对接受该方案移植的患者密切监测HHV-6病毒载量。

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引用本文的文献

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Stem Cells Transl Med. 2016 Mar;5(3):350-7. doi: 10.5966/sctm.2015-0199. Epub 2016 Jan 21.
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Comparison of the Effects of Different Cryoprotectants on Stem Cells from Umbilical Cord Blood.
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Stem Cells Int. 2016;2016:1396783. doi: 10.1155/2016/1396783. Epub 2015 Dec 7.