HLA-haploidentical transplantations for primary immunodeficiencies: A single-center experience
HLA-haploidentical transplantations for primary immunodeficiencies: A single-center experience
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DOI：
10.1111/j.1399-3046.2012.01703.x
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发表时间：
2012-08-01
影响因子：
1.3
通讯作者：
Ikinciogullari, Aydan
中科院分区：
文献类型：
--
作者：
Cipe, Funda Erol;Dogu, Figen;Ikinciogullari, Aydan
[10] Cipe FE，Dogu F，Aytekin C，Yuksek M，Kendirli T，Yildiran A，博兹多甘G，卡拉塔斯D，Reisli I，Dalva K，Arpaci F，Ikinciogullari A. HLA半相合移植治疗原发性免疫缺陷：一项单中心经验翻译后摘要：严重联合免疫缺陷的特点是T和B淋巴细胞的深刻缺陷。造血干细胞移植是目前唯一能治愈SCID患儿的治疗方法。本文报告了18例患者（15例SCID，2例Omenn综合征和1例MHC II类缺陷）的30例HLA半相合移植的临床特征和结局。诊断时患者的年龄范围为1.5至9个月（中位数：4个月）。从诊断到首次移植的中位时间为1个月。输注的CD 34+干细胞剂量范围为7 - 94.2 x 106/kg。18例患者中有9例在诊断时发现CMV抗原血症阳性;因此，他们都没有接受预处理方案。最常见的并发症是移植失败（61%），因此在7名患者中进行了重复移植（2 - 4次）。淋巴移植的平均时间为17.5天（中位数：16，范围：1129天）。15例SCID患者中有10例（67%）存活，具有稳定的完全供体嵌合体。然而，所有三名非SCID患者均死亡。总之，在没有匹配的家庭供体的情况下，来自父母供体的HLA-半相合移植代表了一种现成的治疗选择，特别是对于SCID患者，提供了很高的治愈机会。
Cipe FE, Dogu F, Aytekin C, Yuksek M, Kendirli T, Yildiran A, Bozdogan G, Karatas D, Reisli I, Dalva K, Arpaci F, Ikinciogullari A. HLA-haploidentical transplantations for primary immunodeficiencies: A single-center experience. Abstract: SCID is characterized by profound deficiencies of T and B lymphocytes. HSCT is the only curative treatment for children with SCID. The clinical characteristics and outcome of 30 HLA-haploidentical transplantations in 18 patients (15 SCID, two Omenn syndrome, and one MHC Class II deficiency) are reported here. The age of patients at diagnosis ranged from one and half to nine months (median: four months). The median time was one month between the diagnosis and the time of the initial transplantation. Infused CD34+ stem cell dose was ranged between 7 and 94.2 x 106/kg. Nine of 18 patients were found to be positive for CMV antigenemia at diagnosis; therefore, none of them received a conditioning regimen. The most common complication was graft failure (61%), so repeated transplantations (two to four) were performed in seven patients. The mean time of lymphoid engraftment was 17.5 days (median: 16, range: 1129 days). Ten of 15 SCID (67%) patients survived with a stable complete donor chimerism. However, all three non-SCID patients died. In conclusion, in the absence of a matched family donor, HLA-haploidentical transplantation from parental donors represents a readily available treatment option especially for patients with SCID, offering a high chance of cure.