Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML):: results of the EWOG-MDS/EBMT trial
Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML):: results of the EWOG-MDS/EBMT trial
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DOI：
10.1182/blood-2004-05-1944
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发表时间：
2005-01-01
期刊：
影响因子：
20.3
通讯作者：
Niemeyer, CM
中科院分区：
文献类型：
--
作者：
Locatelli, F;Nöllke, P;Niemeyer, CM
异基因造血干细胞移植（HSCT）是治疗青少年粒单核细胞白血病（JMML）的唯一有效方法。我们，欧洲儿童MDS工作组（EWOG-MDS）和欧洲血液和骨髓移植（EBMT）组，报告了100例JMML儿童（67例男孩和33例女孩）在接受包括白消安、环磷酰胺和美法仑在内的准备方案后接受未经操作的HSCT的结局。48名和52名儿童分别接受了来自HLA相同亲属或无关供体（LID）的移植。造血干细胞来源分别为骨髓79例、外周血14例、脐带血7例。24例患儿术前均行脾切除术。移植相关死亡率和白血病复发的5年累积发生率分别为13%和35%。年龄大于4岁预示着疾病复发的风险增加。亲属或UD接受HSCT的儿童的5年无事件生存率分别为55%和49%（P = NS），存活患者的中位观察时间为40个月（范围，6至144）。在多变量分析中，年龄大于4岁和女性预测预后较差。这项研究的结果与以前发表的报告相比，有利。疾病复发仍然是治疗失败的主要原因。UD-HSCT受者的结果与接受HLA相同同胞移植的儿童相当。(C)2005年，美国血液学会。
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only proven curative therapy for juvenile myelomonocytic leukemia (JMML). We, the European Working Group on Childhood MDS (EWOG-MDS) and the European Blood and Marrow Transplantation (EBMT) Group, report the outcome of 100 children (67 boys and 33 girls) with JMML given unmanipulated HSCT after a preparative regimen including busulfan, cyclophosphamide, and melphalan. Forty-eight and 52 children received transplants from an HLA-identical relative or an unrelated donor (LID), respectively. The source of hematopoietic stem cells was bone marrow, peripheral blood, and cord blood in 79,14, and 7 children, respectively. Splenectomy had been performed before HSCT in 24 children. The 5-year cumulative incidence of transplantation-related mortality and leukemia recurrence was 13% and 35%, respectively. Age older than 4 years predicted an increased risk of disease recurrence. The 5-year probability of event-free survival for children given HSCT from either a relative or a UD was 55% and 49%, respectively (P = NS), with median observation time of patients alive being 40 months (range, 6 to 144). In multivariate analysis, age older than 4 years and female sex predicted poorer outcome. Results of this study compare favorably with previously published reports. Disease recurrence remains the major cause of treatment failure. Outcome of UD-HSCT recipients is comparable to that of children receiving transplants from an HLA-identical sibling. (C) 2005 by The American Society of Hematology.