单倍型相合造血干细胞移植与同胞全相合供者移植治疗阵发性睡眠性血红蛋白尿的比较
[Comparison of haploidentical hematopoietic stem cell transplantation and matched-sibling donor transplantation for the treatment of paroxysmal nocturnal hemoglobinuria].
作者信息
Liu L M, Zhou H F, Wang Q Y, Qiu H Y, Tang X W, Han Y, Fu C C, Jin Z M, Chen S N, Sun A N, Miao M, Wu D P
机构信息
The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology, Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Collaborative Innovation Center of Hematology, Suzhou 215006, China.
出版信息
Zhonghua Xue Ye Xue Za Zhi. 2019 Apr 14;40(4):306-311. doi: 10.3760/cma.j.issn.0253-2727.2019.04.008.
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) . 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. There were no differences in terms of gender, age, patients of PNH-AA and median time from diagnosis to transplantation between the 2 groups (>0.05) . The median values of absolute mononuclear cell counts and CD34 cells infused were 10.74 (4.80-22.86) ×10/kg and 12.19 (5.14-17.25) ×10/kg (=0.866) , 3.57 (0.68-7.80) ×10/kg and 4.00 (3.02-8.42) ×10/kg (=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 (=0.065) , 19 (range, 11-75) and 13 (range, 11-25) days (=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 (=0.294) , the incidences of grade Ⅰ-Ⅳ acute graft-versus-host disease (aGVHD) were 32.0% and 20.0% (=0.343) , grade Ⅱ-Ⅳ aGVHD were 16.0%, 13.3% (=0.759) , chronic GVHD were 30.7% and 24.6% (=0.418) , moderate-severe chronic GVHD were 12.7% and 7.1% (=0.522) respectively, in haplo-HSCT and MSD-HSCT groups. The incidences of infection were 32.0% (8/25) and 26.7% (4/15) (=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) % (=0.520) , GVHD-free and failure-free survival (GFFS) were (78.3±8.6) % and (92.9±6.9) % (=0.250) respectively, in haplo-HSCT and MSD-HSCT groups. 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.
比较单倍体相合供者造血干细胞移植（haplo-HSCT）与同胞全相合供者移植（MSD-HSCT）治疗阵发性睡眠性血红蛋白尿（PNH）的疗效。回顾性分析2007年7月至2018年5月期间接受HSCT的40例PNH患者（haplo-HSCT组25例，MSD-HSCT组15例）的临床资料，比较haplo-HSCT组和MSD-HSCT组的疗效。两组在性别、年龄、PNH-AA患者及从诊断到移植的中位时间方面无差异（>0.05）。haplo-HSCT组和MSD-HSCT组输注的绝对单核细胞计数和CD34细胞的中位值分别为10.74（4.80-22.86）×10⁸/kg和12.19（5.14-17.25）×10⁸/kg（P=0.866），3.57（0.68-7.80）×10⁶/kg和4.00（3.02-8.42）×10⁶/kg（P=0.151）。所有患者均实现完全植入，无患者发生移植失败。haplo-HSCT组和MSD-HSCT组髓系和血小板植入的中位持续时间分别为12天（范围9-26天）和11天（范围7-15天）（P=0.065），19天（范围11-75天）和13天（范围11-25天）（P=0.027）。haplo-HSCT组中位随访26个月（4-65个月），MSD-HSCT组中位随访36个月（4-132个月）（P=0.294），haplo-HSCT组和MSD-HSCT组Ⅰ-Ⅳ级急性移植物抗宿主病（aGVHD）的发生率分别为32.0%和20.0%（P=0.343），Ⅱ-Ⅳ级aGVHD分别为16.0%、13.3%（P=0.759），慢性GVHD分别为30.7%和24.6%（P=0.418），中重度慢性GVHD分别为12.7%和7.1%（P=0.522）。haplo-HSCT组和MSD-HSCT组感染发生率分别为32.0%（8/25）和26.7%（4/15）（P=1.000）。无患者发生早期死亡和复发。haplo-HSCT组和MSD-HSCT组的三年总生存率（OS）估计分别为（86.5±7.3）%和（93.3±6.4）%（P=0.520），无移植物抗宿主病和无失败生存率（GFFS）分别为（78.3±8.6）%和（92.9±6.9）%（P=0.250）。初步结果表明，haplo-HSCT是PNH的一种可行选择，疗效良好，haplo-HSCT和MSD-HSCT产生相似的治疗效果。
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