Reduced-intensity conditioning hematopoietic SCT for pediatric patients with LAD-1: clinical efficacy and importance of chimerism
Reduced-intensity conditioning hematopoietic SCT for pediatric patients with LAD-1: clinical efficacy and importance of chimerism
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DOI：
10.1038/bmt.2011.140
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发表时间：
2012-05-01
影响因子：
4.8
通讯作者：
Ghavamzadeh, A.
中科院分区：
文献类型：
--
作者：
Hamidieh, A. A.;Pourpak, Z.;Ghavamzadeh, A.
患有白细胞粘附缺陷i型（ladi）的儿科患者会经历严重和复发性的危及生命的细菌感染，并伴有脓形成失败和伤口愈合延迟。ladi是一种罕见的遗传性疾病，由白细胞CD18整合素表达突变引起，导致白细胞，特别是嗜中性粒细胞通过血管内间隙粘附和迁移缺陷。造血SCT是ladi患者唯一有效的治疗选择。自2007年以来，在一项前瞻性试验中，我们为10名连续转介至本中心的ladi患者制定了低强度调节方案。根据现有数据，这是第一次有如此多的ladi患者接受这种方案的治疗。本研究试图表明，降低强度的治疗方案对ladi患者有良好的效果，甚至对那些有合并症的患者也是如此。移植后，一些患者出现混合嵌合，然而，在我们的研究中，混合嵌合并没有发生移植排斥反应。骨髓移植（2012）47,646-650；doi: 10.1038 / bmt.2011.140;2011年7月11日在线发布
Pediatric patients with leukocyte adhesion deficiency type-I (LAD-I) experience severe and recurrent life-threatening bacterial infections with failure of pus formation and delayed wound healing. LAD-I is a rare inherited disease caused by mutation in the leukocyte CD18 integrin expression, resulting in defective adherence and migration of leukocytes, in particular neutrophilic granulocytes through the intravascular space. Hematopoietic SCT is the only curative treatment option available to patients with LAD-I. Since 2007, in a prospective trial, reduced-intensity conditioning regimen have been developed for 10 consecutive patients with LAD-I who were referred to our center. Based upon available data, it is the first time that such a number of patients affected by LAD-I have been treated with this regimen. This study attempts to show that reduced-intensity regimen leads to a favorable result in LAD-I patients even in those who have experienced comorbid complications. Following transplantation, some patients develop mixed chimerism, however, in our study mixed chimerism was not followed by transplant rejection. Bone Marrow Transplantation (2012) 47, 646-650; doi: 10.1038/bmt.2011.140; published online 11 July 2011