异基因造血干细胞移植治疗儿童血液病

Allogeneic Hemepoietic Stem Cell Transplantation for Treatment of Children Hematological Diseases

目的造血干细胞移植可以根治白血病、再生障碍性贫血、血红蛋白病及先天性免疫缺陷等疾病,通过临床观察,分析研究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。采用白消安、环磷酰胺或环磷酰胺、全身照射为基础的预处理的方案。结果14例患者植入,1例于移植后15 d死亡,未达植入标准。白细胞的植入时间各组间没有明显差异;脐血移植血小板的植入明显较骨髓或外周血延迟(P<0.05)。主要并发症为巨细胞病毒感染和复发,11例患者生存;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%。结论脐血移植具有移植物抗宿主疾病轻、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。提出了儿童脐血移植的选择策略。

Objective To observe the effect of allogeneic stem cell transplantation for treatment of children hematological diseases. Methods Fifteen children suffered from leukemia, lymphoma, myelodysplastic syndrome, and severe aplastic anemia were treated with stem cell transplantation, including 3 sibling related cord blood transplants （CBT）, 9 unrelated CBT, 2 related sibling peripheral hemopoietic stem cell transplants and 1 bone marrow transplant. The conditioning regimens were based on Busulphan/Cyclophosphamide or Cyclophosphamide/Total body irradiation protocols. Results Fourteen children were successfully engrafted and 1 failed due to early viral 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 cytomegalovirus （CMV） infections and relapse. Up to now 11 children were alive and 10 were event free survival. The longest survival time is 6 years. Kaplanmeier survival curve predicts that 1 year survival is 76. 6M and 5 year survival is 57. 4%. Conclusion CBT has a broad and prospective application in children hematological diseases with the advantages of less incidence of graft-versus-host disease （GVHD）, easier to control and to find transplant. The selective strategy for CBT is proposed.