Haploidentical hematopoietic stem cell transplant in paroxysmal nocturnal hemoglobinuria
Haploidentical hematopoietic stem cell transplant in paroxysmal nocturnal hemoglobinuria
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DOI：
10.3109/10428194.2015.1068309
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发表时间：
2016-04-02
影响因子：
2.6
通讯作者：
Wu, Depei
中科院分区：
文献类型：
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作者：
Tian, Hong;Liu, Liming;Wu, Depei
18例阵发性夜间血红蛋白尿（PNH）患者接受同种异体造血干细胞移植（alloo - hsct），这些患者来自hla -单倍体相同的供体（HRD, n = 10）或hla匹配的供体（n = 5来自兄弟姐妹，n = 3来自无血缘关系的供体）。一例在非相关供体造血干细胞移植后出现原发性移植物衰竭。他接受了第二次HRD-HSCT，但在造血功能恢复后死于巨细胞病毒肺炎。其他17例患者实现了持续移植和全供体嵌合。HRD-HSCT组中有4例发生II/III级急性移植物抗宿主病（aGVHD）， hla匹配HSCT组中有5例发生II级aGVHD。在所有18例患者中，10例发生慢性GVHD (cGVHD)，只有1例接受HRD-HSCT的患者发生广泛的cGVHD。最后随访时，HRD-HSCT组9例存活，hla匹配HSCT组全部存活且不依赖输血。我们的研究结果表明，同种异体造血干细胞移植是治疗PNH的一种很有希望的方法，而HRD-HSCT对于缺乏hla匹配供体的PNH患者来说是一种可行的选择。
Eighteen patients with paroxysmal nocturnal hemoglobinuria (PNH) receiving allogeneic hematopoietic stem cell transplant (allo-HSCT), either from HLA-haploidentical donors (HRD; n = 10) or HLA-matched donors (n = 5 from siblings and n = 3 from unrelated donors), were retrospectively evaluated. One showed primary graft failure following unrelated-donor HSCT. He was given a second HRD-HSCT, but died from cytomegalovirus pneumonia after achieving hematopoietic recovery. The other 17 patients achieved sustained engraftment and full-donor chimerism. Four in the HRD-HSCT group experienced grade II/III acute graft-versus-host disease (aGVHD), and five in the HLA-matched HSCT group developed grade II aGVHD. Among all 18 patients, 10 developed chronic GVHD (cGVHD), only one patient receiving HRD-HSCT developed extensive cGVHD. Nine in the HRD-HSCT group and all those in the HLA-matched HSCT group were alive and transfusion-independent at last follow-up. Our findings suggest that allo-HSCT is a promising treatment for PNH, and HRD-HSCT is a viable option for patients with PNH who lack HLA-matched donors.