非清髓异基因造血干细胞移植治疗阵发性睡眠性血红蛋白尿2例并文献复习

Successful application of nonmyeloablative stem cell transplantation in two paroxysmal nocturnal hemoglobinuria patients and literature review

目的:探讨非清髓异基因造血干细胞移植(NST)治疗阵发性睡眠性血红蛋白尿(PNH)的疗效。方法:采用HLA全相合的同胞外周血造血干细胞移植,经环磷酰胺(CTX)、福达拉滨(Flud)和抗人淋巴细胞免疫球蛋白(ALG)方案进行NST预处理,环孢素A(CsA)、甲氨喋呤(MTX)和霉酚酸酯(MMF)三联预防移植物抗宿主病(GVHD)。结果:病例1在确诊后就进行了NST,输注CD34+细胞3.51×106/kg(按受者体重计算),第28天成功植入,无严重的GVHD表现及并发症;病例2在PNH确诊17年后进行NST,输注CD34+细胞数量2.2×106/kg,第60天成功植入,分别出现急性和慢性GVHD。结论:同胞全相合的NST是治疗PNH的有效方法,在PNH诊断早期进行NST以及回输足量的CD34+细胞是NST的有益因素。

Objective：This study was aimed to evaluate the effect of nonmyeloablative stem cell transplantation （NST） in paroxysmal nocturnal hemoglobinuria （PNH）.Method：NST was performed with conditioning regimen consisting of cyclophosphamide,anti-lymphocyte globulin and fludarabine. The patients received peripheral blood stem cells from their human leukocyte antigen-matched siblings. Prophylaxis against graft-vs-host disease was performed with cyclosporine A,mycophenolate mofetil and methotrexate.Result：The results showed that the 30-year-old patient received NST after diagnosis as PNH. The grafts contained 3.51×106 CD34＋ cells per kilogram of body weight. No meaningful regimen-related toxicities of this patient were documented. Donor chimerism was achieved on day 28 and thereafter. The other 40-year-old patient with 17-years disease course of PNH also received NST. The grafts contained 2.2×106 CD34＋ cells per kilogram of body weight. Donor chimerism was achieved on day 60 and thereafter. Acute and chronic GVHD were developed after NST.Conclusion：It is concluded that NST may become a safe and curative approach in patients with PNH. Performing NST at the early stage after diagnosis and receiving enough CD34＋ cells will be beneficial to successful NST.