同胞与非血缘脐血移植治疗儿童恶性血液病的效果分析

背景:近年来,脐血逐渐成为亲缘及非血缘骨髓或外周血造血干细胞移植的一种极其关键的替代干细胞来源,被越来越多地用于儿童恶性血液病的治疗。目的:比较同胞与非血缘脐血移植治疗儿童恶性血液病的临床疗效。方法:回顾性分析1998-01-01/2018-12-31于郑州大学第一附属医院造血干细胞移植中心接受同胞脐血移植及非血缘脐血移植治疗儿童恶性血液病患者的临床资料,所有脐血移植患者均采用清髓性预处理方案,同时应用环孢素A±吗替麦考酚酯方案预防移植物抗宿主病。结果与结论:①2例同胞脐血移植患者及3例非血缘脐血移植患者造血植入失败继发感染死亡,其他全部脐血移植患者均顺利达到造血植入;同胞脐血移植组、非血缘脐血移植组中性粒细胞与血小板中位植入时间分别为17 d(11-43 d),18 d(12-45 d),P=0.307]与20.5 d(15-50 d),27 d(18-56 d),P=0.773],差异均无显著性意义;②同胞脐血移植组、非血缘脐血移植组急性移植物抗宿主病与慢性移植物抗宿主病的发生率分别为(36%vs.43%,P=0.737)与(15%vs.33%,P=0.412),差异均无显著性意义;同胞脐血移植组与非血缘脐血移植组移植后感染的发生率为56%,71%,差异无显著性意义(P=0.343);③同胞脐血移植组、非血缘脐血移植组2年总体生存率与2年无复发生存率分别为(61%vs.36%,P=0.301)与(56%vs.33%,P=0.151),差异均无显著性意义;同胞脐血移植组、非血缘脐血移植组5年总体生存率和5年无复发生存率分别为(54%vs.24%,P=0.044)与(50%vs.20%,P=0.039),两组在长期生存方面差异有显著性意义;④结果显示同胞与非血缘脐血移植均是治疗儿童恶性血液病安全有效可行的移植方式,尤其在儿童血液病患者替代供者移植的长期生存方面明显受益。

Matched-sibling donor versus unrelated umbilical cord blood transplantation for treating hematological malignancies in children

BACKGROUND:In recent years,umbilical cord blood has gradually become a crucial alternative source of stem cells for related and unrelated bone marrow or peripheral blood hematopoietic stem cell transplantation,which is increasingly used in the treatment of hematological malignancies in children.OBJECTIVE:To compare the clinical efficacy of sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation for treating hematological malignancies in children.METHODS:The clinical data of children with hematological malignancies who received umbilical cord blood transplantation at the Hematopoietic Stem Cell Transplantation Center of the First Affiliated Hospital of Zhengzhou University between January 1,1998 and December 31,2018 was retrospectively analyzed.All the patients received myelablative conditioning regimen,and cyslosporine A combined with or without mycophenolate mofetil were concurrently adopted for graft-versus-host disease prophylaxis.RESULTS AND CONCLUSION:(1)Two patients in the sibling donor umbilical cord blood transplantation group and three in the unrelated umbilical cord blood transplantation group did not attain hematological engraftment and subsequently died from infection,and other patients succeeded in hematological engraftment.The median time of neutrophil and platelet engraftment in the sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation groups was17 days(11-43 days),18 days(12-45 days),P=0.307]and20.5 days(15-50 days),27 days(18-56 days),P=0.773].There was no significant difference between the two groups.(2)The incidence of acute graft-versus-host disease and chronic graft-versus-host disease in the sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation groups was 36%vs.43%(P=0.737)and 15%vs.33%(P=0.412).There was no significant difference between the two groups.There was also no significant difference in the incidence of infection after transplantation between sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation groups(56%vs.71%,P=0.343).(3)There were no significant differences in the 2-year overall survival(61%vs.36%,P=0.301),or 2-year relapse-free survival(56%vs.33%,P=0.151).The 5-year overall survival and 5-year relapse-free survival in the sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation groups were 54%vs.24%(P=0.044)and 50%vs.20%(P=0.039).The results showed that there was a significant difference in long-term survival rate between two groups.(4)Our results reveal that both sibling donor umbilical cord blood transplantation and unrelated umbilical cord blood transplantation are safe,effective and applicable for children with hematological malignancies.In particular,there are significant benefits in the long-term survival of substitute donor transplantation for pediatric patients with hematological malignancies.