Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Collaborative Innovation Center of Hematology, The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology, Suzhou, China.
Department of Laboratory Medicine, Affiliated Hospital of University of Electronic Science and Technology of China, Sichuan Academy of Medical Sciences and Sichuan Provincial People's Hospital, Chengdu, China.
Biol Blood Marrow Transplant. 2019 Aug;25(8):1544-1549. doi: 10.1016/j.bbmt.2019.02.024. Epub 2019 Mar 8.
We analyzed the outcomes of 44 patients with paroxysmal nocturnal hemoglobinuria (PNH) who received allogeneic hematopoietic stem cell transplantation (allo-HSCT) (haploidentical [haplo]-donors, 25; matched sibling donors [MSDs], 15; and matched unrelated donors, 4) between July 2007 and May 2018. All patients achieved successful donor engraftment. The median time was 12 days (range, 7 to 26) for myeloid engraftment and 13 days (range, 11 to 75) for platelets. The cumulative incidences were 15.91% and 2.27% for grades II to IV and grades III to IV acute graft-versus-host disease (GVHD), respectively, with a median follow-up time of 36 months (range, 4 to 132). The cumulative incidences were 26.73% for chronic GVHD and 9.70% for moderate to severe chronic GVHD. No patients relapsed. The probabilities of 3-year overall survival (OS) and GVHD-free, failure-free survival (GFFS) were 90.4% ± 4.6% and 85.6% ± 5.4%, respectively. The 3-year OS rates of the haplo-donor and MSD groups were 86.5% ± 7.3% versus 93.3% ± 6.4% (P = .520). The 3-year GFFS rates of the haplo-donor and MSD groups were 78.3% ± 8.6% versus 92.9% ± 6.9% (P = .250). The preliminary results indicated that allo-HSCT is a feasible option for patients with PNH; however, this should not be considered as a first-choice therapy, because the results seemed to only suggest rather than confirm that haplo-HSCT and MSD-HSCT exerted similar therapeutic efficacies.
我们分析了 2007 年 7 月至 2018 年 5 月期间 44 例阵发性睡眠性血红蛋白尿症（PNH）患者接受异基因造血干细胞移植（allo-HSCT）（单倍体[haplo]-供体 25 例，匹配的同胞供体 15 例，匹配的无关供体 4 例）的结果。所有患者均成功获得供体植入。髓系植入的中位时间为 12 天（范围为 7 至 26 天），血小板植入的中位时间为 13 天（范围为 11 至 75 天）。Ⅱ至Ⅳ级和Ⅲ至Ⅳ级急性移植物抗宿主病（GVHD）的累积发生率分别为 15.91%和 2.27%，中位随访时间为 36 个月（范围为 4 至 132 个月）。慢性 GVHD 的累积发生率为 26.73%，中重度慢性 GVHD 的累积发生率为 9.70%。无患者复发。3 年总生存率（OS）和无 GVHD、无失败生存（GFFS）的累积发生率分别为 90.4%±4.6%和 85.6%±5.4%。haplo 供体和 MSD 组的 3 年 OS 率分别为 86.5%±7.3%和 93.3%±6.4%（P=0.520）。haplo 供体和 MSD 组的 3 年 GFFS 率分别为 78.3%±8.6%和 92.9%±6.9%（P=0.250）。初步结果表明，allo-HSCT 是 PNH 患者可行的选择；然而，这不应该被视为首选治疗，因为结果似乎只是表明而不是证实haplo-HSCT 和 MSD-HSCT 具有相似的治疗效果。
