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重型地中海贫血造血干细胞移植后100天内针对不稳定混合嵌合体的第二次小型移植。

The second mini-transplant for unstable mixed chimerism within the first 100 days after hematopoietic stem cell transplant in severe thalassemia.

作者信息

Choeyprasert Worawut, Pakakasama Samart, Anurathapan Usanarat, Songdej Duantida, Sirachainun Nongnuch, Sirireung Somtawin, Panthangkool Wanpen, Hongeng Suradej

机构信息

Department of Pediatrics, Mahidol University, Bangkok, Thailand.

出版信息

Pediatr Transplant. 2012 Sep;16(6):E238-40. doi: 10.1111/j.1399-3046.2011.01577.x. Epub 2011 Sep 5.

DOI:10.1111/j.1399-3046.2011.01577.x
PMID:21895905
Abstract

Allogeneic HSCT is the only curative treatment for severe thalassemia disease. MC occurs in one-third of these patients within the first two months after HSCT; this is a major risk factor of graft rejection, especially when RHCs are more than 25%. There is still no consensus for the management of MC, especially in the early phase of HSCT. The DLI has also been described in the treatment of MC following HSCT for hemoglobinopathies, but its success is still not guaranteed. The second HSCT has been an approach used in an attempt to cure patients who reject their graft. Concern about toxicity of conditioning regimen, the second HSCT is usually delayed for at least a year after the first HSCT. We would like to demonstrate the successful use of the second mini-allogeneic HSCT in hemoglobin E/β-thalassemia with evidence of unstable MC in the first 100 days after allogeneic HSCT to prevent further graft loss after allogeneic HSCT.

摘要

异基因造血干细胞移植(HSCT)是重型地中海贫血疾病的唯一治愈性治疗方法。移植后血细胞嵌合体(MC)在这些患者中三分之一会在HSCT后的头两个月内出现;这是移植物排斥的主要危险因素,尤其是当红细胞嵌合体(RHCs)超过25%时。对于MC的管理,尤其是在HSCT的早期阶段,仍然没有共识。在血红蛋白病患者HSCT后的MC治疗中也描述了供体淋巴细胞输注(DLI),但其成功率仍无保障。第二次HSCT一直是试图治愈移植物排斥患者所采用的一种方法。由于担心预处理方案的毒性,第二次HSCT通常在第一次HSCT后至少推迟一年。我们想展示第二次小剂量异基因HSCT在血红蛋白E/β地中海贫血患者中的成功应用,这些患者在异基因HSCT后的前100天有不稳定MC的证据,以防止异基因HSCT后进一步的移植物丢失。

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