Objective: To compare the outcomes between haploidentical donor hematopoietic stem cell transplantation (haplo-HSCT) and matched-sibling donor transplantation (MSD-HSCT) for paroxysmal nocturnal hemoglobinuria (PNH) . Methods: The clinical data of 40 PNH patients received HSCT (haplo-HSCT=25, MSD-HSCT=15) from July 2007 to May 2018 were analyzed retrospectively to compare the outcomes between haplo-HSCT and MSD-HSCT groups. Results: There were no differences in terms of gender, age, patients of PNH-AA and median time from diagnosis to transplantation between the 2 groups (P>0.05) . The median values of absolute mononuclear cell counts and CD34+ cells infused were 10.74 (4.80-22.86) ×108/kg and 12.19 (5.14-17.25) ×108/kg (P=0.866) , 3.57 (0.68-7.80) ×106/kg and 4.00 (3.02-8.42) ×106/kg (P=0.151) respectively, in haplo-HSCT and MSD-HSCT groups. All patients attained complete engraftment, no patient occurred graft failure. The median durations for myeloid and platelet engraftment were 12 (range, 9-26) and 11 (range, 7-15) days (P=0.065) , 19 (range, 11-75) and 13 (range, 11-25) days (P=0.027) respectively, in haplo-HSCT and MSD-HSCT groups. During a median follow-up of 26 (4-65) months in haplo-HSCT and 36 (4-132) months in MSD-HSCT groups (P=0.294) , the incidences of grade Ⅰ-Ⅳ acute graft-versus-host disease (aGVHD) were 32.0% and 20.0% (P=0.343) , grade Ⅱ-Ⅳ aGVHD were 16.0%, 13.3% (P=0.759) , chronic GVHD were 30.7% and 24.6% (P=0.418) , moderate-severe chronic GVHD were 12.7% and 7.1% (P=0.522) respectively, in haplo-HSCT and MSD-HSCT groups. The incidences of infection were 32.0% (8/25) and 26.7% (4/15) (P=1.000) respectively, in haplo-HSCT and MSD-HSCT groups. No patients occurred early death and relapse. Three-year estimated overall survival (OS) were (86.5±7.3) % and (93.3 ±6.4) % (P=0.520) , GVHD-free and failure-free survival (GFFS) were (78.3±8.6) % and (92.9±6.9) % (P=0.250) respectively, in haplo-HSCT and MSD-HSCT groups. Conclusion: The preliminary results indicated that haplo-HSCT was a feasible choice for PNH with favorable outcomes, haplo-HSCT and MSD-HSCT produced similar therapeutic efficacy.
目的： 比较单倍型与同胞全相合移植治疗阵发性睡眠性血红蛋白尿症（PNH）的疗效及安全性。 方法： 总结2007年7月至2018年5月40例（单倍型移植25例，同胞全相合移植15例）接受异基因造血干细胞移植的PNH患者临床资料，对两组疗效及安全性进行比较。 结果： 单倍型移植组及同胞全相合移植组患者性别、年龄、PNH-再生障碍性贫血（AA）患者比例、诊断至移植时间差异均无统计学意义（P>0.05）。单倍型移植组及同胞全相合组回输单个核细胞数（MNC）分别为10.74（4.80~22.86）×108/kg与12.19（5.14~17.25）×108/kg（P=0.866），回输CD34+细胞数分别为3.57（0.68~7.80）×106/kg与4.00（3.02~8.42）×106/kg（P=0.151）。所有患者均成功植入，单倍型移植组及同胞全相合移植组粒细胞植入时间分别为12（9~26）d与11（7~15）d（P=0.065），血小板植入时间分别为19（11~75）d与13（11~25）d（P=0.027）。单倍型移植组与同胞全相合移植组Ⅰ~Ⅳ度急性GVHD发生率分别32.0%、20.0%（P=0.343），Ⅱ~Ⅳ度急性GVHD发生率分别为16.0%、13.3%（P=0.759）。慢性GVHD发生率分别为30.7%、24.6%（P=0.418），中重度慢性GVHD发生率分别为12.7%、7.1%（P=0.522）。移植过程中单倍型移植组与同胞全相合移植组感染发生率分别为32.0%（8/25）、26.7%（4/15）（P=1.000），未发生早期死亡。单倍型移植组及同胞全相合移植组预期3年总生存（OS）率分别为（86.5±7.3）%、（93.3±6.4）%（P=0.520），无移植物抗宿主病无失败生存（GFFS）率分别为（78.3± 8.6）%、（92.9 ± 6.9）%（P=0.250）。在随访期内两组患者均无复发。 结论： 单倍型移植用于治疗PNH具有与同胞全相合移植相似的疗效及安全性，在无全相合供者情况下可作为PNH患者有价值的治疗手段。.
Keywords: Hematopoietic stem cell transplantation; Hemoglobinuria, paroxysmal; Treatment outcome.