National Clinical Research Center for Hematologic Disease, Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.
Department of Hematology, Guangzhou First People's Hospital, School of Medicine, South China University of Technology, Guangzhou, China.
J Hematol Oncol. 2019 Sep 2;12(1):87. doi: 10.1186/s13045-019-0775-9.
Haploidentical transplantation has been proposed as an effective treatment for severe aplastic anemia (SAA). The majority of patients have more than one HLA-haploidentical donor. Herein, we compared the outcomes between different donor-recipient relationships for optimal haploidentical donor selection in acquired SAA.
We conducted a multicenter study based on a registered database of 392 patients with SAA treated with allogeneic hematopoietic stem cell transplantation (allo-HSCT) between 2006 and 2018. In total, 223 patients received grafts from father donors, 47 from mother donors, 91 from siblings, 29 from children, and 2 from collateral donors.
Of the 381 patients who survived more than 28 days, 379 (99.5%) recipients were engrafted. The 2-year overall survival (OS) was 86.6 ± 2.5%, 87.1 ± 4.9%, 84.3 ± 3.9%, and 92.2 ± 5.1% for recipients of father, mother, sibling, and child grafts, respectively, (P = 0.706). The 2-year failure-free survival (FFS) was 82.8 ± 2.7%, 86.7 ± 5.1%, 80.8 ± 4.2%, and 92.5 ± 5.1% for recipients of father, mother, sibling, and child grafts, respectively, (P = 0.508). There was no difference in the incidence of either acute or chronic graft-versus-host disease (GVHD) among the different donor sources in multivariate analyses. There were also no differences in the OS or FFS among the different donor sources in the Cox regression analysis. However, OS was significantly better in the patients with a shorter history of aplastic anemia (< 12 months), better performance status (ECOG scores 0-1), or moderate graft mononuclear cell (MNC) counts (6-10 × 10/kg), and in female recipients with male donors. The FFS was also higher in patients with a shorter history of aplastic anemia (< 12 months) and better performance status (ECOG scores 0-1).
Fathers, mothers, siblings, and children are all suitable haploidentical donors for patients with SAA.
单倍体相合移植已被提出作为治疗严重再生障碍性贫血（SAA）的有效方法。大多数患者有一个以上的 HLA 单倍体供者。在此，我们比较了不同供受者关系之间的结果，以优化获得性 SAA 中单倍体相合供者的选择。
我们进行了一项多中心研究，基于 2006 年至 2018 年间接受异基因造血干细胞移植（allo-HSCT）治疗的 392 例 SAA 患者的注册数据库。共有 223 例患者接受父亲供者的移植物，47 例接受母亲供者的移植物，91 例接受同胞供者的移植物，29 例接受子女供者的移植物，2 例接受旁系供者的移植物。
在 381 例存活超过 28 天的患者中，379 例（99.5%）受者植入成功。父亲、母亲、同胞和子女供者的 2 年总生存率（OS）分别为 86.6±2.5%、87.1±4.9%、84.3±3.9%和 92.2±5.1%（P=0.706）。父亲、母亲、同胞和子女供者的 2 年无失败生存率（FFS）分别为 82.8±2.7%、86.7±5.1%、80.8±4.2%和 92.5±5.1%（P=0.508）。多因素分析显示，不同供者来源之间急性或慢性移植物抗宿主病（GVHD）的发生率无差异。Cox 回归分析也显示，不同供者来源之间的 OS 或 FFS 无差异。然而，在史较短的再生障碍性贫血（<12 个月）、表现状态较好（ECOG 评分 0-1）或中等移植物单核细胞（MNC）计数（6-10×10/kg）的患者中，OS 明显较好，在女性受者中，男性供者的 OS 也较好。史较短的再生障碍性贫血（<12 个月）和表现状态较好（ECOG 评分 0-1）的患者的 FFS 也较高。
父亲、母亲、同胞和子女均为 SAA 患者的合适单倍体相合供者。
