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Abstract：
Traditionally, bone marrow (BM) has been preferred as a source of stem cells (SCs) in pediatric hematopoietic SC transplantation (HSCT); however, the use of peripheral blood SCs (PBSC) has recently increased. With advancing graft-versus-host disease (GVHD) prophylaxis, whether the BM is still a better SC source than PB in sibling donor HSCT remains controversial. Here, we compared the results of BM transplantation (BMT) and PBSC transplantation (PBSCT) in pediatric patients with malignant or non-malignant diseases receiving sibling HSCT using a total of 7.5 mg/kg of anti-thymocyte globulin (ATG). We retrospectively reviewed children who received HSCT from a sibling donor between 2005 and 2020 at Seoul National University Children\'s Hospital. Of the 86 patients, 40 underwent BMT, and 46 underwent PBSCT. Fifty- six patients had malignant diseases, whereas thirty patients had non-malignant diseases. All conditioning regimens comprised ATG. Busulfan-based myeloablative conditioning regimens were administered to patients with malignant diseases and approximately half of those with non-malignant diseases. The remaining half of the patients with non-malignant diseases were administered cyclophosphamide-based reduced- intensity conditioning regimens. According to studies conducted at our center, all BM donors received G-CSF before harvest to achieve early engraftment. In all 86 patients (47 males and 39 females), the median age at the time of HSCT was 11.4 (range, 0.7 - 24.6) years. The median follow-up period was 57.9 (range, 0.9-228.6) months, and the corresponding values for those with BM and PBSC were 77 (range, 2.4-228.6) months and 48.7 (range, 0.9-213.2) months, respectively. Engraftment failure occurred in one patient with BM and no patient with PBSC. The cumulative incidence of acute GVHD with grades II-IV was higher in PBSC (BM 2.5%, PBSC 26.1%, p = 0.002), but there was no significant difference in those with grades III-IV acute GVHD (BM 0%, PBSC 6.5%, p = 0.3703) and extensive chronic GVHD (BM 2.5%, PBSC 11.6%, p = 0.1004). There were no significant differences in treatment-related mortality (TRM) (BM 14.2%, PBSC 6.8%, p = 0.453), 5-year event-free survival (EFS) (BM 71.5%, PBSC 76.2%, p = 0.874), and overall survival (OS) rates (BM 80.8%, PBSC 80.3%, p = 0.867) between BM and PBSC in the univariate analysis. In the multivariate analysis, which included all factors with p < 0.50 in the univariate analysis, there was no significant prognostic factor for EFS or OS. There was no significant difference in the relapse incidence between BM and PBSC among patients with malignant diseases (BM 14.2%, PBSC 6.8%, p = 0.453). Additionally, there were no significant differences in the TRM, 5-year EFS, and OS rates between malignant and non-malignant diseases nor between the busulfan-based myeloablative regimen and reduced-intensity chemotherapy using cyclophosphamide. In this study, we showed no significant differences in EFS, OS, TRM, and GVHD, except for acute GVHD grades II-IV, between BMT and PBSCT from sibling donors, using ATG (a total of 7.5 mg/kg). Therefore, PB collection, which is less invasive for donors and less labor-intensive for doctors, could also be considered an acceptable SC source for sibling donor HSCT in children.
摘要：
传统上,骨髓（BM）在儿科造血干细胞移植（HSCT）中被优选为干细胞（SCs）的来源；然而，最近外周血SCs（PBSC）的使用有所增加。随着移植物抗宿主病（GVHD）预防的推进，在同胞供体HSCT中，BM是否仍然是比PB更好的SC来源仍存在争议。这里,我们比较了BM移植(BMT)和PBSC移植(PBSCT)在患有恶性或非恶性疾病的儿科患者中使用总计7.5mg/kg的抗胸腺细胞球蛋白(ATG)进行HSCT的结果.我们回顾性分析了2005年至2020年在首尔国立大学儿童医院接受同胞捐献者HSCT的儿童。86名患者中,40人接受了BMT，46例接受PBSCT。56名患者患有恶性疾病，而30例患者患有非恶性疾病。所有的调节方案包括ATG。对恶性疾病患者和大约一半的非恶性疾病患者进行了基于白消安的清髓性预处理方案。剩余的一半患有非恶性疾病的患者施用基于环磷酰胺的降低强度的调节方案。根据我们中心的研究,所有BM供体在收获前接受G-CSF,以实现早期植入.在所有86例患者中（男性47例，女性39例），HSCT时的中位年龄为11.4岁(范围，0.7-24.6）年。中位随访期为57.9（范围，0.9-228.6）个月，BM和PBSC的相应值为77(范围，2.4-228.6)个月和48.7(范围，0.9-213.2）个月，分别。植入失败发生在一名BM患者和无PBSC患者中。在PBSC中，II-IV级急性GVHD的累积发生率较高（BM2.5％，PBSC26.1%，p=0.002),但是III-IV级急性GVHD患者没有显着差异（BM0％，PBSC6.5%，p=0.3703)和广泛的慢性GVHD(BM2.5%，PBSC11.6%，p=0.1004）。治疗相关死亡率（TRM）没有显着差异（BM14.2％，PBSC6.8%，p=0.453),5年无事件生存率（EFS）（BM71.5%，PBSC76.2%，p=0.874),和总生存率（OS）（BM80.8%，PBSC80.3%，单变量分析中BM和PBSC之间的p=0.867）。在多变量分析中，其中包括单变量分析中p<0.50的所有因素，EFS或OS没有显著的预后因素。恶性疾病患者中BM和PBSC的复发率没有显着差异（BM14.2％，PBSC6.8%，p=0.453)。此外,TRM没有显着差异，5年EFS,恶性和非恶性疾病之间的OS率，以及基于白消安的清髓方案和使用环磷酰胺的低强度化疗之间的OS率。在这项研究中,我们在EFS上没有显着差异，操作系统,TRM,和GVHD,除了急性GVHDII-IV级,来自同胞捐赠者的BMT和PBSCT之间，使用ATG（总共7.5mg/kg）。因此,PB集合,这对捐赠者来说侵入性较小，对医生来说劳动强度较小，也可以被认为是儿童同胞供者HSCT可接受的SC来源。
