异基因造血干细胞移植治疗儿童血液病

Allogeneic hematopoietic stem cell transplantation for treatment of childhood hematological diseases

目的观察异基因造血干细胞移植对儿童血液病的治疗效果。方法采用异基因造血干细胞移植治疗15例儿童血液病,其中非血缘脐血移植9例,同胞脐血移植3例,同胞外周血干细胞移植2例,同胞骨髓移植1例。采用马利兰/环磷酰胺(BU/CY)或环磷酰胺/全身照射(CY/TBI)为基础的预处理的方案。结果14例患儿植入成功,白细胞的植入时间各组间差异无统计学意义;脐血移植血小板的植入时间较骨髓或外周血延迟(P<0.05)。主要并发症为CMV感染和复发,11例患儿生存,其中10例无病存活(占66.7%),存活最长时间为6年。Kaplan-meier生存曲线提示:1年生存率为76.6%,预计5年生存率为57.4%。结论脐血移植具有GVHD轻且较容易控制、搜寻时间短等优点,对儿童患者具有广泛的应用前景。

Objectives To observe the effect of allogeneie hematopoietic stem cell transplantation for treatment of children with hematological diseases. Methods Fifteen children suffered from leukemia, lymphoma, myelodysplastie syndrome, and severe aplastie anemia were carried out stem cell transplantation, including 3 siblings donated cord blood transplants （CBT）, 9 unrelated CBT, 2 related sibling peripheral hemopoietie stem cell transplants and 1 bone marrow transplant. The conditioning regimens were based on Busulphan/Cyelophosphamide or Cyclophosphamide/Total body irradiation protocols. Results Engraftment was successful in 14 children and 1 failed due to early virus infection. The engraftment of white blood cells in CBT group was comparable to that in peripheral stem cell transplants or bone marrow transplant, but the platelets engraftment was significantly delayed （P 〈 0.05）. The main complications were eytomegalovirus infection and relapse. Up to now 11 children were alive and 10 were event free survival. The longest survival time is 6 years. Kaplan-meier survival curve predicts that 1 year survival is 76.6% and 5 year survival is 57.4%. Conclusions CBT has a broad and prospective application in childhood hematological diseases for the advantages of less graft-versus-host disease, easy control and the shorter time from search to transplant. The selective strategy for CBTis proposed.