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有关β地中海贫血造血干细胞移植的具体问题

Author
Emanuele Angelucci, MD
Section Editor
Robert S Negrin, MD
Deputy Editor
Jennifer S Tirnauer, MD
Translators
李庆山, 主任医师

引言

纯合子β-地中海贫血患者需要长期输血才能维持生存；然而这些长期输血患者如果不持续接受去铁治疗，最终会因铁超负荷而死亡。尽管在人类中使用基因治疗进行了第一次尝试，但是造血干细胞移植(hematopoietic cell transplantation, HCT)仍然是该疾病唯一广泛可用的治愈性治疗方法。

在HCT方面，β-地中海贫血患者的特点使临床处理不同于白血病患者的临床处理。这些特点包括：

●组织损害，特别是与铁超负荷相关的肝脏损害

●缺乏既往化疗和免疫抑制

●缺乏恶性克隆

                    

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Literature review current through: 2017-06 . | This topic last updated: 2015-06-23.
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