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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。
Objective To investigate the therapeutic effect of allogeneic hematopoietic stem cell transplantation (allo-HSCT) on children with chronic myelogenous leukemia (CML) and to find out the possible influencing factors in order to improve the prognosis of the patients. Methods Twenty cases of CML patients treated with allo-HSCT were divided into 4 groups according to age, sex, diagnosis interval to transplantation, matched HLA matching of recipients, disease status of children at transplantation, and acute and chronic graft versus host disease gost-v-host disease, GVHD) and other factors for efficacy analysis. Results As of the follow-up date, of the 20 patients, 13 survived without disease and 7 died, 4 of them died of acute severe GVHD, 2 died of chronic GVHD and its complications, 1 died of graft-versus-host disease, 3 The overall disease-free survival rate was (64.6 ± 1.1%). Univariate analysis showed that age was one of the most important factors influencing the prognosis of children with CML (P <0.05). Aged ≥10 years old was a poor prognostic factor for CML pediatric transplantation. Other factors, including gender, HLA typing, disease at transplantation Status, diagnosis to transplantation interval and acute and chronic GVHD had no significant effect on prognosis (P> 0.05). Multivariate logistic regression analysis further proved that only age was the prognostic factor (P <0.01). A variety of severe acute and chronic GVHD is the most important cause of death in patients. Select the full complement of 10 donors for transplantation for treatment of good prognosis. Conclusions Allo-HSCT can effectively treat CML in children. Allo-HSCT should be performed in children with CML older than 10 years old. Allo-HSCT should be selected as the best donor for transplant. The prevention and treatment of GVHD should be improved. Children after transplantation treatment outcome.