Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China
Turk J Haematol. 2022 Jun 1;39(2):117-129. doi: 10.4274/tjh.galenos.2022.2022.0084. Epub 2022 Apr 22.
We retrospectively compared the outcomes of patients with severe aplastic anemia (SAA) who received haploidentical hematopoietic stem cell transplantation (haplo-HSCT) combined or not combined with umbilical cord-derived mesenchymal stem cells (UC-MSCs).
A total of 101 patients with SAA were enrolled in this study and treated with haplo-HSCT plus UC-MSC infusion (MSC group, n=47) or haplo-HSCT alone (non-MSC group, n=54).
The median time to neutrophil engraftment in the MSC and non-MSC group was 11 (range: 8-19) and 12 (range: 8-23) days, respectively (p=0.049), with a respective cumulative incidence (CI) of 97.82% and 97.96% (p=0.101). Compared to the non-MSC group, the MSC group had a lower CI of chronic graft-versus-host disease (GVHD) (8.60±0.25% vs. 24.57±0.48%, p=0.048), but similar rates of grades II-IV acute GVHD (23.40±0.39% vs. 24.49±0.39%, p=0.849), grades III-IV acute GVHD (8.51±0.17% vs. 10.20±0.19%, p=0.765), and moderate-severe chronic GVHD (2.38±0.06% vs. 7.45±0.18%, p=0.352) were observed. The estimated 5-year overall survival (OS) rates were 78.3±6.1% and 70.1±6.3% (p=0.292) while the estimated 5-year GVHD-free, failure-free survival (GFFS) rates were 76.6±6.2% and 56.7±6.9% (p=0.045) in the MSC and non-MSC groups, respectively.
In multivariate analysis, graft failure was the only adverse predictor for OS. Meanwhile, graft failure, grades III-IV acute GVHD, and moderate-severe chronic GVHD could predict worse GFFS. Our results indicated that haplo-HSCT combined with UC-MSCs infusion was an effective and safe option for SAA patients.
我们回顾性比较了接受单倍体造血干细胞移植(haplo-HSCT)联合或不联合脐带间充质干细胞(UC-MSCs)治疗的重型再生障碍性贫血(SAA)患者的结局。
本研究共纳入 101 例 SAA 患者,接受 haplo-HSCT 联合 UC-MSC 输注(MSC 组,n=47)或 haplo-HSCT 单独治疗(非 MSC 组,n=54)。
MSC 组和非 MSC 组中性粒细胞植入的中位时间分别为 11(范围:8-19)和 12(范围:8-23)天(p=0.049),累积发生率(CI)分别为 97.82%和 97.96%(p=0.101)。与非 MSC 组相比,MSC 组慢性移植物抗宿主病(GVHD)的 CI 较低(8.60±0.25% vs. 24.57±0.48%,p=0.048),但 2 级-4 级急性 GVHD(23.40±0.39% vs. 24.49±0.39%,p=0.849)、3 级-4 级急性 GVHD(8.51±0.17% vs. 10.20±0.19%,p=0.765)和中重度慢性 GVHD(2.38±0.06% vs. 7.45±0.18%,p=0.352)的发生率相似。MSC 组和非 MSC 组的 5 年总生存率(OS)分别为 78.3±6.1%和 70.1±6.3%(p=0.292),5 年无 GVHD-复发生存(GFFS)率分别为 76.6±6.2%和 56.7±6.9%(p=0.045)。
多变量分析显示,移植物失败是 OS 的唯一不良预测因素。同时,移植物失败、3 级-4 级急性 GVHD 和中重度慢性 GVHD 可预测更差的 GFFS。我们的研究结果表明,haplo-HSCT 联合 UC-MSCs 输注是治疗 SAA 患者的一种有效且安全的选择。