Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea.
Environmental Health Center for Childhood Leukemia and Cancer, Chonnam National University Hwasun Hospital, Hwasun, Korea.
J Korean Med Sci. 2020 Feb 24;35(7):e46. doi: 10.3346/jkms.2020.35.e46.
This study aimed to assess the outcome of stem cell transplantation (SCT), including overall survival (OS), failure-free survival (FFS) and graft-versus-host disease (GvHD)-free/failure-free survival (GFFS), and to analyze prognostic factors in children with aplastic anemia (AA).
From 1991 to 2018, 43 allogeneic SCT recipients were enrolled in the study to investigate the demographic characteristics, survival outcomes and prognostic factors.
With the median follow-up of 7.1 years, the estimated 10-year OS, FFS, GFFS were 86.0%, 60.5%, and 51.2%, respectively. Matched related donors (MRD, n = 28) showed better 10-year OS than unrelated donors (n = 15) (96.4% vs. 66.7%; = 0.006). Engraftment failure was seen in 13 patients (30.2%). Donor-type aplasia was seen in 13.8% (4/29) after fludarabine (Flu)-based conditioning (Flu-group), while in 42.6% (6/14) after cyclophosphamide (Cy)-based regimen (Cy-group) ( = 0.035). Six patients died. The 10-year OS in Cy-group was 92.9% (n = 14, all MRD), while that of Flu-group was 82.1% (n = 29; = 0.367). But Flu-group tended to have better FFS and GFFS than Cy-group, although Flu-group had less MRDs (41.4% vs. 100%; = 0.019), and higher proportion of previous immunosuppressive treatment (IST; 62% vs. 21.4%, = 0.012). In MRD transplants, OS was similar between Flu-group (100%, n = 14) and Cy-group (92.9%, n = 14), while FFS (100.0% vs. 42.9%; = 0.001) and GFFS (85.7% vs. 35.7%; = 0.006) were significantly better in Flu-group. Stem cell sources, irradiation in the conditioning, and method of GvHD prophylaxis did not significantly influence the outcome.
This study reviewed SCT outcomes for pediatric AA with changes of transplant strategies over the last 25 years. The FFS and GFFS were higher in Flu-group than in Cy-group, especially in matched related transplantation. Graft failure including donor-type aplasia remains troublesome even with Flu-based conditioning. Further refinement of transplant strategies to ensure better quality-of-life should be pursued.
本研究旨在评估干细胞移植（SCT）的结果，包括总生存率（OS）、无失败生存率（FFS）和移植物抗宿主病（GvHD）-无失败生存率（GFFS），并分析再生障碍性贫血（AA）患儿的预后因素。
1991 年至 2018 年，共纳入 43 例异基因 SCT 受者，以研究其人口统计学特征、生存结果和预后因素。
中位随访 7.1 年后，估计 10 年 OS、FFS、GFFS 分别为 86.0%、60.5%和 51.2%。在匹配的亲缘供者（MRD，n=28）中，10 年 OS 优于非亲缘供者（n=15）（96.4%比 66.7%；=0.006）。13 例患者出现植入失败（30.2%）。在氟达拉滨（Flu）为基础的预处理（Flu 组）中，13.8%（4/29）出现供者型再生障碍，而在环磷酰胺（Cy）为基础的方案（Cy 组）中，有 42.6%（6/14）出现供者型再生障碍（=0.035）。6 例患者死亡。Cy 组的 10 年 OS 为 92.9%（n=14，均为 MRD），而 Flu 组为 82.1%（n=29；=0.367）。但 Flu 组的 FFS 和 GFFS 均优于 Cy 组，尽管 Flu 组的 MRD 较少（41.4%比 100%；=0.019），且既往免疫抑制治疗（IST）的比例较高（62%比 21.4%；=0.012）。在 MRD 移植中，Flu 组（n=14）和 Cy 组（n=14）的 OS 相似，而 Flu 组的 FFS（100.0%比 42.9%；=0.001）和 GFFS（85.7%比 35.7%；=0.006）显著优于 Cy 组。干细胞来源、预处理中的照射以及 GvHD 预防方法对结果没有显著影响。
本研究回顾了过去 25 年中，干细胞移植治疗儿科再生障碍性贫血的结果，结果显示，与 Cy 组相比，Flu 组的 FFS 和 GFFS 更高，尤其是在匹配相关移植中。即使采用 Flu 为基础的预处理，仍存在供者型再生障碍的植入失败问题。应进一步完善移植策略，以确保更高的生活质量。
