异基因造血干细胞移植治疗阵发性睡眠性血红蛋白尿症

目的 探讨异基因造血干细胞移植（allo-HSCT）治疗阵发性睡眠性血红蛋白尿症（PNH）的临床效果及安全性.方法 回顾性分析4例接受allo-HSCT的患者资料,其中3例为难治性PNH,1例为PNH-再生障碍性贫血综合征,均为HLA 6/6相合亲缘供者.预处理均采用白消安（BU）/环磷酰胺（CY）＋抗人T淋巴细胞兔球蛋白（ATG-F）方案,急性移植物抗宿主病（GVHD）预防采用环孢素A（CsA）＋甲氨蝶呤（MTX）,并复习相关文献.结果 移植后4例患者经DNA短串联重复序列多态性分析证明均为完全供者植入,中性粒细胞＞ 0.5×109/L的平均时间为15d（11～18d）,血小板计数＞ 20×109/L的平均时间为19 d（14～28 d）.移植后15～30 d CD55、CD59恢复正常.2例发生Ⅱ度急性GVHD,1例发生慢性GVHD,未发生移植排斥.中位随访时间14个月（7～42个月）,患者均无病生存.结论 allo-HSCT是根治难治性PNH的有效方法.

Exploration on the treatment of paroxysmal nocturnal hemoglobinuria by allogeneic hematopoietic stem cell transplantation

Objective To explore the efficacy and safety of allogeneic hematopoietic stem cell transplantation （allo-HSCT） in the treatment of refractory paroxysmal nocturnal hemoglobinuria （PNH）.Methods The clinical data of four cases including three patients with refractory PNH and one patient with PNH-aplastic anemia syndrome who underwent allo-HSCT with HLA 6/6 family donor either were analyzed retrospectively.Conditioning regimen was BU/CY ＋ ATG scheme,combining cyclosporin and methotrexate to prevent acute graft versus host disease （aGVHD）.The treatment of the cases was discussed with literatures review.Results All patients＇ deoxyribonucleic acid （DNA） were totally donors＇ by DNA polymorphism analysis of short tandem repeat sequence.The number of neutrophil was more than 0.5×109/L in 15 days （11-18 days） averagely after transplantation,and platelet was more than 20×109/L in 19 days （14-28 days）.The expression of CD55 and CD59 on the membrane of erythrocyte and neutrophil returned to normal on 15-30 days after transplantation.In addition,aGVHD at grade 2 occurred in two cases,and chronic GVHD in one case,excitingly,no graft rejection happened.All recipients were followed up for 14 months （7-42 months）with disease-free survival.Conclusion allo-HSCT is an effective technique to cure refractory PNH.