Objective: To analyze the clinical efficacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for paroxysmal nocturnal hemoglobinuria (PNH), and preliminarily explore the role of an improved post-transplantation cyclophosphamide (PTCy) based conditioning regimen in PNH patients receiving transplantation.
Methods: Clinical related data of PNH sufferers receiving allo-HSCT in Department of Hematology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology were collected, and hematopoietic reconstitution, chimerism, PNH cloning, graft-versus-host disease (GVHD), infection, and survival were analyzed.
Results: Totally five PNH patients receiving allo-HSCT were enrolled, including 1 case with classic PNH, 3 cases with aplastic anemia-PNH syndrome, 1 case with myelodysplastic syndrome, three of them (case 1-3) received the improved PTCy based conditioning regimen before HSCT. All sufferers engrafted successfully within 28 days, the median time of neutrophil and platelet engraftment was 11 days and 12 days, respectively, no patient occurred acute or chronic GVHD, after a median follow-up of 16 months, all recipients survived and completely eliminated PNH cloning.
Conclusion: Allo-HSCT can completely clear PNH cloning and restore hematopoietic function with controllable complications, and the improved PTCy based conditioning regimen is proved to be effective in PNH transplantation.
题目: 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: 分析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）的临床疗效及安全性，同时初步探索以改良后置环磷酰胺（PTCy）为基础的预处理方案在PNH移植中的应用.
方法: 收集华中科技大学同济医学院附属同济医院接受allo-HSCT治疗的PNH患者的临床资料，分析移植后造血重建、嵌合率、PNH克隆、移植物抗宿主病、感染和患者生存情况.
结果: 5例接受allo-HSCT治疗的PNH患者中，经典型PNH 1例，再生障碍性贫血-PNH综合征3例，PNH合并骨髓增生异常综合征1例，其中3例（病例1－3）采用改良PTCy为基础的预处理方案，所有患者均在28 d内植入成功，中性粒细胞和血小板的中位植入时间分别为11 d和12 d，无急、慢性移植物抗宿主病发生，中位随访时间16个月，所有患者均存活且完全清除PNH克隆.
结论: Allo-HSCT能够完全清除PNH患者体内的异常克隆，重建正常造血，具有良好的临床疗效，同时，以改良PTCy为基础的预处理方案在PNH的移植治疗中行之有效.