单份非血缘脐血移植治疗幼年型粒单核细胞白血病的疗效分析

Efficacy Analysis of Unrelated Cord Blood Transplantation in the Treatment of Juvenile Myelomonocytic Leukemia

目的:探讨非血缘脐血移植(UCBT)治疗幼年型粒单核细胞白血病(JMML)的临床疗效和安全性。方法:回顾性分析2011年10月至2019年7月5例接受单份UCBT的JMML患儿的临床资料。5例患儿均为男性,中位发病年龄1.5(0.4-5.0)岁。全部患儿均采用清髓性不含ATG的预处理方案,并予以环孢素A联合霉酚酸酯方案预防移植物抗宿主病(GVHD)。结果:5例患者中4例成功植入,1例原发性植入失败接受挽救性相合造血单倍体干细胞移植。2例Ⅲ-Ⅳ度急性GVHD均有效控制,无慢性GVHD发生。3例患儿无病生存,无1例复发。结论:UCBT是治疗JMML的有效方法。

Objective:To explore the clinical efficacy and safety of unrelated umbilical cord blood transplantation(UCBT)in the treatment of Juvenile myelomonocytic leukemia(JMML).Methods:The clinical data of 5 children with JMML who were treated with unrelated UCBT from October 2011 to July 2019 were retrospectively analyzed.The age of onset for the five children(male)ranged from 0.4 to 5.0 years old,with a median age of 1.5 years old.All the patients received myeloablative conditioning regimen without ATG to whom cyclosporine A(CsA)with shortterm mycophenolate mofetil(MMF)was given for GVHD prophylaxis.Results:Four children acquired engraftment.One patient received secondary haploidentical hematopoietic stem cell transplantation because of the failure in the first unrelated UCBT.GradeⅢtoⅣaGVHD occurred in 2 cases and was controlled,and none of the patients developed cGVHD.Three cases achieved long-time disease free survival,and no patient relapsed.Conclusion:UCBT is an effective treatment for children with JMML.