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Abstract：
The application of haploidentical hematopoietic stem cell transplantation (HSCT) with mesenchymal stem cell (MSC) infusion as a treatment regimen for severe aplastic anemia (SAA) has been reported to be efficacious in single-arm trials. However, it is difficult to assess without comparing the results with those from a first-line, matched-sibling HSCT. Herein, we retrospectively reviewed 91 patients with acquired SAA. They received HSCT from haploidentical donors combined with MSC transfer (HID group). We compared these patients with 103 others who received first-line matched-sibling HSCT (MSD group) to evaluate relative treatment efficacy. Compared with the patients in the MSD group, those in the HID group presented with higher incidences of grades II-IV and III-IV acute graft versus host disease (aGvHD) and chronic graft versus host disease (cGvHD) (p < 0.05). However, the incidence of myeloid and platelet engraftment, graft failure, poor graft function, and extensive cGvHD were comparable for both groups. The median follow-up was 36.6 months and the 3-year overall survival rate was similar for both groups (83.5% versus 79.1%). Univariate and multivariate analyses revealed that time intervals greater than 4 months from diagnosis to transplantation, experienced graft failure, poor graft function, or grade III-IV aGvHD were significantly associated with adverse outcomes. All HID patients received MSC co-transplantation with hematopoietic stem cells. However, the infused MSCs were derived from umbilical cord (UC-MSC group; 43 patients) or bone marrow (BM-MSC group; 48 patients) and were administered at different medical centers. We first compared the outcomes between the two groups and detected that the BM-MSC group exhibited lower incidences of grade III-IV aGvHD and cGvHD (p < 0.05). This study suggests that co-transplantation of hematopoietic and MSCs significantly reduces the risk and incidence of graft rejection and may effectively improve overall survival in patients with SAA even in the absence of closely related histocompatible donor material.
摘要：
据报道，在单臂试验中，使用间充质干细胞（MSC）输注的单倍体造血干细胞移植（HSCT）作为重度再生障碍性贫血（SAA）的治疗方案是有效的。然而,如果不将结果与一线的结果进行比较，就很难进行评估，匹配的兄弟HSCT。在这里，我们回顾性分析了91例获得性SAA患者.他们接受来自单倍体供体的HSCT结合MSC转移(HID组)。我们将这些患者与其他103名接受一线配对同胞HSCT（MSD组）的患者进行了比较，以评估相对治疗效果。与MSD组患者相比，HID组中II-IV级和III-IV级急性移植物抗宿主病(aGvHD)和慢性移植物抗宿主病(cGvHD)的发病率较高(p<0.05).然而,骨髓和血小板植入的发生率，移植失败，移植物功能差，和广泛的cGvHD在两组中具有可比性。中位随访时间为36.6个月，两组的3年总生存率相似（83.5%对79.1%）。单变量和多变量分析显示，从诊断到移植的时间间隔大于4个月，经历过移植失败，移植物功能差，或III-IV级aGvHD与不良结局显著相关.所有HID患者均接受MSC与造血干细胞的共移植。然而,输注的MSCs来自脐带(UC-MSC组;43例患者)或骨髓(BM-MSC组;48例患者),并在不同的医疗中心给药.我们首先比较了两组之间的结果，并发现BM-MSC组表现出III-IV级aGvHD和cGvHD的发生率较低（p<0.05）。这项研究表明，造血和MSC的共移植显着降低了移植物排斥的风险和发生率，并且即使在没有密切相关的组织相容性供体材料的情况下，也可以有效地改善SAA患者的总体生存率。
