Department of Pediatrics, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Department of Pediatrics, State Key Laboratory of Experimental Haematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Blood Cells Mol Dis. 2024 Jan;104:102793. doi: 10.1016/j.bcmd.2023.102793. Epub 2023 Aug 25.
Unrelated umbilical cord blood transplantation (UCBT) for bone marrow failure (BMF) disorders using conditioning regimens without Anti-Thymocyte Globulin (ATG) has been used as an alternative transplantation for emerging patients without matched-sibling donors. Experience with this transplant modality in children is limited, especially as a secondary treatment for transplant failure patients.
We retrospectively reviewed 17 consecutive bone marrow failure patients who underwent unrelated umbilical cord blood transplantation in our center and received conditioning regimens of Total Body Irradiation (TBI) or Busulfan (BU) + Fludarabine (FLU) + Cyclophosphamide (CY).
Among the 17 BMF patients, 15 patients were treated with first cord blood transplantation and another 2 with secondary cord blood transplantation because of graft failure after first haploidentical stem cell transplantation at days +38 and +82. All patients engrafted with a median donor cell chimerism of 50 % at days +7 (range, 16 %-99.95 %) and finally rose to 100 % at days +30. Median time to neutrophil engraftment was 19 days (range, 12-30) and time to platelet engraftment was 32 days (range, 18-61). Pre-engraftment syndrome (PES) was found in 16 patients (94.11 %, 16/17). Cumulative incidence of grades II to IV acute GVHD was 58.8 % (95 % CI: 32.7-84.9 %), and 17.6 % (95 % CI: 2.6-37.9 %) of patients developed chronic GVHD. The 3-year overall survival (OS) and failure-free survival (FFS) rates were 92.86 ± 6.88 %.
UCBT is an effective alternative treatment for bone marrow failure pediatric patients. TBI/BU + FLU + CY regimen ensure a high engraftment rate for unrelated umbilical cord blood transplantation, which overcomes the difficulty of graft failure. Secondary salvage use of cord blood transplantation may still be useful for patients who have failed after other transplantation.
对于骨髓衰竭（BMF）疾病，采用不含抗胸腺细胞球蛋白（ATG）的预处理方案进行无关脐血移植（UCBT），已作为无匹配同胞供体的新兴患者的替代移植方法。在儿童中，这种移植方式的经验有限，尤其是作为移植失败患者的二线治疗。
我们回顾性分析了 17 例在我院接受非相关脐血移植的骨髓衰竭患者，预处理方案为全身照射（TBI）或白消安（BU）+氟达拉滨（FLU）+环磷酰胺（CY）。
在 17 例 BMF 患者中，15 例患者接受了首次脐血移植，另外 2 例患者因首次单倍体造血干细胞移植后第 38 天和第 82 天发生移植物失败而接受了二次脐血移植。所有患者均植入供者细胞嵌合体，中位数为 50%（范围为 16%-99.95%），最终于第 30 天上升至 100%。中性粒细胞植入中位时间为 19 天（范围为 12-30 天），血小板植入中位时间为 32 天（范围为 18-61 天）。16 例患者（94.11%，16/17）出现植入前综合征（PES）。Ⅱ至Ⅳ度急性移植物抗宿主病（GVHD）累积发生率为 58.8%（95%可信区间：32.7%-84.9%），17.6%（95%可信区间：2.6%-37.9%）的患者发生慢性 GVHD。3 年总生存率（OS）和无失败生存率（FFS）分别为 92.86%±6.88%。
UCBT 是治疗儿童骨髓衰竭的有效替代方法。TBI/BU+FLU+CY 方案确保了无关脐血移植的高植入率，克服了移植物失败的困难。对于其他移植后失败的患者，二次使用脐血移植可能仍然有用。
