Abstract：
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.
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.
摘要：
背景：使用不含抗胸腺细胞球蛋白（ATG）的预处理方案治疗骨髓衰竭（BMF）疾病的非相关脐带血移植（UCBT）已被用作没有匹配同胞供体的新兴患者的替代移植。这种儿童移植方式的经验有限，特别是作为移植失败患者的二次治疗。
方法：我们回顾性回顾了17例连续骨髓衰竭患者，这些患者在我们中心接受了无关的脐带血移植，并接受了全身照射（TBI）或白消安（BU）+氟达拉滨（FLU）+环磷酰胺（CY）的预处理方案。
结果：在17例BMF患者中，在第38天和第82天，由于首次单倍体干细胞移植后的移植失败，15例患者接受了首次脐带血移植，另外2例接受了二次脐带血移植。所有患者在第+7天移植的中位供体细胞嵌合率为50%(范围,16%-99.95%)，最后在+30天上升到100%。中性粒细胞植入的中位时间为19天(范围，12-30），血小板植入时间为32天（范围，18-61).在16例患者中发现了植入前综合征（PES）（94.11％，16/17).II级至IV级急性GVHD的累积发生率为58.8%（95%CI：32.7-84.9%），17.6%(95%CI: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%。
结论：UCBT是骨髓衰竭儿科患者的有效替代治疗方法。TBI/BU+FLU+CY方案确保无关脐带血移植的高植入率，克服了移植失败的困难。脐带血移植的二次抢救使用可能对其他移植后失败的患者仍然有用。
方法：我们回顾性回顾了17例连续骨髓衰竭患者，这些患者在我们中心接受了无关的脐带血移植，并接受了全身照射（TBI）或白消安（BU）+氟达拉滨（FLU）+环磷酰胺（CY）的预处理方案。
结果：在17例BMF患者中，在第38天和第82天，由于首次单倍体干细胞移植后的移植失败，15例患者接受了首次脐带血移植，另外2例接受了二次脐带血移植。所有患者在第+7天移植的中位供体细胞嵌合率为50%(范围,16%-99.95%)，最后在+30天上升到100%。中性粒细胞植入的中位时间为19天(范围，12-30），血小板植入时间为32天（范围，18-61).在16例患者中发现了植入前综合征（PES）（94.11％，16/17).II级至IV级急性GVHD的累积发生率为58.8%（95%CI：32.7-84.9%），17.6%(95%CI: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%。
结论：UCBT是骨髓衰竭儿科患者的有效替代治疗方法。TBI/BU+FLU+CY方案确保无关脐带血移植的高植入率，克服了移植失败的困难。脐带血移植的二次抢救使用可能对其他移植后失败的患者仍然有用。
