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

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

摘要:

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（P<0.05），年龄≥10岁是CML儿童移植治疗预后不良的因素；其他因素，包括性别、HLA配型、移植时疾病状态、诊断至移植间隔时间以及急、慢性GVHD等均对治疗预后无明显影响（P>0.05）。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。

Abstract:

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.MethodsThe 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).ResultsAt 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.ConclusionsAllo-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.