摘要目的:探讨单份非血缘脐血移植(UCBT)治疗成人阵发性睡眠性血红蛋白尿症(PNH)的可行性及疗效。方法:回顾性分析2014年5月至2019年12月安徽省立医院血液科7例应用单份UCBT治疗成人PNH受者的临床资料。7例受者中6例重度PNH，1例极重度PNH。原发性PNH 4例，PNH-再生障碍性贫血(AA)综合征3例。男性5例，女性2例，中位年龄29岁(20～47岁)，中位体重60 kg(50～71 kg)，诊断至移植中位时间为62.5个月(7.7～171个月)，全部受者均伴输血依赖。预处理方案：清髓性6例，减低强度1例。预防移植物抗宿主病(GVHD)方案均为环孢素A联合短程吗替麦考酚酯，均未使用抗胸腺细胞免疫球蛋白(ATG)。输入脐血有核细胞的中位数为2.4×10 7/kg(1.71～4.28×10 7/kg)，其中输入CD34+细胞中位数为1.58×10 5/kg(0.88～3.02×10 5/kg)。 结果:所有受者均获得髓系及红系植入，中性粒细胞绝对计数≥0.5×10 9/L的中位时间17 d(15～21 d)，网织红细胞计数≥1%的中位时间27 d(22～45 d)。6例受者获得血小板植入，血小板≥20×10 9/L中位时间37 d(25～101 d)，血小板≥50×10 9/L中位时间62 d(27～157 d)。100 d急性GVHD发生率28.6%(95%CI 0～55.3%)，其中2例Ⅱ度急性GVHD，仅1例受者发生局限慢性GVHD。全部受者中位随访时间13.5个月(3～71.4个月)，6例受者存活，1例PNH-AA综合征伴铁过载受者死于消化道出血。2年总存活率为83.3%(95%CI 27.3～97.5%)。 结论:单份UCBT治疗PNH，可获得很好的植入率及存活率，PNH-AA综合征伴铁过载可能是影响患者预后的重要因素之一。

abstractsObjective:To explore the feasibility and efficacy of umbilical cord blood transplantation (UCBT) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH).Methods:From May 2014 to December 2019, clinical data were retrospectively reviewed for 7 PNH patients undergoing UCBT. The grades were severe ( n=6) and extremely severe ( n=1). The causes were primary PNH ( n=4) and PNH-aplastic anemia (AA) syndrome ( n=3). There were 5 males and 2 females with a median age of 29 (20-47) years, a median weight of 60(50-71) kg and a median time from diagnosis to transplantation of 62.5(7.7-171) months. All of them were accompanied by transfusion dependence. Myeloablative ( n=6) and reduced-intensity ( n=1) pretreatment was offered. The regimen of preventing GVHD was cyclosporine A plus short-term mycophenolate mofetil without ATG. The median number of input nucleated cells was 2.4(1.71-4.28)×10 7/kg and the median number of CD34+ cells 1.58(0.88-3.02)×10 5/kg. Results:Neutrophil and erythroid engraftment was obtained with a median neutrophil engraftment time of 17(15-21) days and a median erythroid engraftment time of 27. Engraftment time of 37(25-101) days for platelets >20×10 9/L and 62(27-157) days for platelets >50×10 9/L. The incidence of 100-day acute GVHD was 28.6%(95%CI 0-55.3%). The severity of GVHD was grade Ⅱ° acute ( n=2) and mild ( n=1). The median follow-up period was 13.5(3-71.4) months. Six patients survived while another with PNH-AA syndrome with iron overload died of gastrointestinal hemorrhage. The 2-year overall survival rate was 83.3%(95%CI 27.3-97.5%). Conclusions:With excellent engraftment and survival in the treatment of PNH, UCBT is indicated for patients without HLA full-match donor. PNH-AA syndrome with iron overload may be one of the important prognostic factors.