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采用宫内输血和无关供者造血细胞移植治疗的纯合子α地中海贫血

Homozygous alpha-thalassemia treated with intrauterine transfusions and unrelated donor hematopoietic cell transplantation.

作者信息

Yi Joanna S, Moertel Christopher L, Baker K Scott

机构信息

Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.

出版信息

J Pediatr. 2009 May;154(5):766-8. doi: 10.1016/j.jpeds.2008.11.031.

Abstract

Recently, intrauterine transfusions and hematopoietic cell transplantation (HCT) have changed homozygous alpha-thalassemia from a frequently fatal disease to a potentially survivable condition. We present a patient with Hemoglobin Bart's disease who was cured after failing to engraft with 1 unrelated HCT, but engrafting after a second unrelated donor HCT.

摘要

最近,宫内输血和造血细胞移植(HCT)已将纯合子α地中海贫血从一种常见的致命疾病转变为一种有可能存活的病症。我们报告了一名患有血红蛋白巴特氏病的患者,该患者在接受1次无关供者造血细胞移植未成功植入,但在接受第2次无关供者造血细胞移植后成功植入并治愈。

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