异基因造血干细胞移植治疗儿童慢性粒细胞性白血病的疗效分析

目的：研究异基因造血干细胞移植（allogeneic hematopoietic stem cell transplantation, allo-HSCT）治疗儿童慢性粒细胞性白血病（chronic myelogenous leukemia, CML）的治疗效果，寻找可能的影响因素，以期改善患者预后。方法：对接受allo-HSCT治疗的20例儿童CML患者，分别从年龄、性别、诊断至移植间隔时间、供受体HLA配型相合情况、移植时患儿疾病状态以及急慢性移植物抗宿主病（gost-v-host disease, GVHD）等多种因素进行疗效分析。结果：截止至随访日期，20例患者中，13例无病存活，7例死亡，其中4例死于急性重度GVHD，2例死于慢性GVHD及其并发症，1例死于移植后复发，3年总无病生存率为（64.6±1.1%）。单因素分析显示年龄是影响儿童CML治疗预后最重要的因素之一（P0.05）。多因素logistic回归分析也进一步证明仅年龄是影响预后的因素（P<0.01）。各种严重急慢性 GVHD是引起患者死亡最重要的原因。选择10位点全相合的供体进行移植治疗预后好。结论：allo-HSCT能有效治疗儿童CML，对于年龄≥10岁的CML患儿宜早期行allo-HSCT移植治疗，且尽可能选择10位点全相合的供体进行移植，积极防治GVHD，改善CML患儿移植治疗后的转归。

Therapeutic efficacy of allogeneic hematopoietic stem cell transplantation in children with chronic myelogenous leukemia

Objective To investigate the therapeutic efficacy of allogeneic hematopoietic stem cell transplantation(allo-HSCT) in children with chronic myelogenous leukemia(CML),and to analyze the possible prognostic factors.Methods The clinical data of 20 children with CML who had received allo-HSCT was analyzed retrospectively to investigate possible prognostic factors,including age,sex,interval between diagnosis and transplantation,HLA matching between donors and recipients,illness status on transplantation and acute and chronic graft-versus-host disease(GVHD).Results At the end of follow-up,13 of the 20 treated children had disease-free survival(DFS) and the rest(7 cases) died.Four died of severe acute GVHD,two of chronic GVHD and its complications,and one of relapse after transplantation.The three-year DFS was(64.6±1.1%).As shown by the univariate analysis,age was the most important prognostic factor in children with CML who had received allo-HSCT(P<0.05),and in children over 10 years,the prognosis was poor.No other of the above factors had a significant impact on prognosis(P>0.05).The multivariate logistic regression analysis also confirmed age as the only prognostic factor(P<0.01).Severe acute and/or chronic GVHD was the most important cause of patient death.10/10 HLA-matched donors could improve the transplantation outcome.Conclusions Allo-HSCT is an effective treatment for children with CML.To improve the prognosis and treatment outcome,children with CML aged over 10 years should receive allo-HSCT as early as possible.10/10 HLA-matched donors are preferred in allo-HSCT and GVHD should be prevented.