Table_1_Comparison of Haploidentical Hematopoietic Stem Cell Transplant With or Without Unrelated Cord Blood Infusion in Severe Aplastic Anemia: Outcomes of a Multicenter Study.docx

The purpose of this study in severe aplastic anemia (SAA) patients was to compare the feasibility and efficacy of haploidentical hematological stem cell transplantation combined with a single unrelated cord blood (UCB) infusion (Haplo-cord-HSCT) or haplo-identical HSCT (Haplo-HSCT) alone. The five-year graft-versus-host disease (GVHD)-free or failure-free survival (GFFS) was similar between the two groups (72.4 ± 3.4% vs. 65.4 ± 5.2%, P = 0.178); however, the five-year overall survival (OS) was more favorable in the Haplo-cord-HSCT group than that in the Haplo-HSCT group (84.0 ± 2.8% vs. 72.6 ± 4.9%, P = 0.022), as was transplantation-related mortality (16.4% vs. 27.4%, P = 0.039). Multivariate analysis showed that Haplo-cord HSCT was the only independent determinant of increased OS (P = 0.013). Explorative subgroup analysis showed that only an Human leukocyte antigen-A (HLA-A) allele match between UCB and the recipient was a beneficial factor for GFFS in the Haplo-cord-HSCT group (P = 0.011). In the haplo-cord with an HLA-A match (n = 139) or mismatch (n = 32) or Haplo-HSCT groups, a haplo-cord HLA-A allele match was associated with lower I–IV and III–IV acute GVHD. The haplo-cord with an HLA-A match subgroup also had higher five-year OS than the Haplo-HSCT group (85.4 ± 3.0% vs. 72.6 ± 4.9%, P = 0.013), and higher five-year GFFS than the Haplo-cord HLA-A allele mismatch subgroup (76.2 ± 3.6% vs. 56.3 ± 8.8%, P = 0.011). These findings suggest that the coinfusion of a single UCB potentially improves survival of Haplo-HSCT in SAA patients and that an HLA-A allele-matched UCB is the preferred option.

本研究针对重型再生障碍性贫血（severe aplastic anemia, SAA）患者，旨在对比单倍体相合造血干细胞移植联合单份无关脐血（unrelated cord blood, UCB）输注（简称Haplo-cord-HSCT）与单纯单倍体相合造血干细胞移植（haplo-identical HSCT, Haplo-HSCT）的可行性与疗效。两组患者的5年无移植物抗宿主病或无失败生存率（graft-versus-host disease-free or failure-free survival, GFFS）相近（72.4±3.4% vs 65.4±5.2%，P=0.178）；但Haplo-cord-HSCT组的5年总生存期（overall survival, OS）优于Haplo-HSCT组（84.0±2.8% vs 72.6±4.9%，P=0.022），移植相关死亡率亦更低（16.4% vs 27.4%，P=0.039）。多因素分析显示，单倍体联合脐血造血干细胞移植是提升总生存期的唯一独立影响因素（P=0.013）。探索性亚组分析表明，在Haplo-cord-HSCT组中，仅脐血与受体间的人类白细胞抗原-A（Human leukocyte antigen-A, HLA-A）等位基因相合是改善GFFS的有利因素（P=0.011）。在HLA-A相合（n=139）或不相合（n=32）的单倍体联合脐血移植组，以及单纯单倍体相合移植组中，HLA-A等位基因相合的单倍体联合脐血移植与更低的I~IV级及III~IV级急性移植物抗宿主病相关。其中，HLA-A相合的单倍体联合脐血移植亚组的5年总生存期亦高于单纯单倍体相合移植组（85.4±3.0% vs 72.6±4.9%，P=0.013），且5年GFFS高于HLA-A等位基因不相合的单倍体联合脐血移植亚组（76.2±3.6% vs 56.3±8.8%，P=0.011）。本研究结果提示，单份无关脐血联合输注可改善重型再生障碍性贫血患者行单倍体相合造血干细胞移植后的生存结局，且人类白细胞抗原-A等位基因相合的脐血为优选方案。