摘要目的 回顾性分析24例慢性髓性白血病(CML)患儿异基因造血干细胞移植(alloHSCT)疗效以及相关影响因素.方法 24例患儿中男16例,女8例,中位年龄12岁(3 ～16岁).其中慢性期(CP)16例;加速期(AP)1例;急变期(BP)7例.所有的病例均诊断为成人型CML,均有Ph染色体,BCR/ABL融合基因移植前均为阳性.预处理方案均采用白消安加环磷酰胺(BUCY)方案.移植方式为HLA配型相合的同胞5例,HLA配型不合的半相合移植14例,非血缘关系外周血干细胞移植5例.移植物抗宿主病(GVHD)预防均采用环孢菌素加短程甲氨蝶呤(MTX)加吗替麦考酚酯(MMF)预防方案.中位随访时间为36个月.结果 (1)所有患者均获得稳定造血重建.24例患儿alloHSCT后死亡4例,3例为急变期行半相合移植,另外1例为慢性期行同胞全合移植术后8个月死于慢性GVHD合并特发性肺炎综合征(IPS),5年总生存率81.0％.(2)不同疾病阶段疗效比较:慢性期患儿16例,死亡1例.加速期患儿1例,存活5年11个月.急变期患儿7例,死亡3例,1例因为复发死于移植后早期(2个月),1例因为复发死于移植后1年,另外1例于移植后3个月死于急性GVHD.(3)不同移植方式疗效比较:24例患儿中,HLA配型全相合5例,死亡1例.亲缘半相合移植14例,死亡3例,均为急变期患儿.非血缘供者5例,全部存活.(4)长期生存(5年以上)患儿10例,3例合并慢性GVHD,其中1例伴有关节挛缩,影响生活质量.结论 alloHSCT治疗儿童慢性粒细胞白血病疗效肯定,供者的选择已经不是异基因移植的障碍,确诊后应早期行异基因造血干细胞移植.预处理方案的调整,移植前后对移植相关合并症有效预防和治疗,对于CML患儿移植后长期高质量生存都有重要意义.

abstractsObjective To retrospectively analyze the effects of allogeneic hematopoietic stem cell transplantation(allo-HSCT) on childhood chronic myelogenous leukemia (CML).Method Of the 24 consecutive cases,16 were boys and 8 were girls.The median age of patients was 12 (3-16) years old; 16 cases were in chronic phase (CP) of CML,1 case in accelerated phase (AP) and 5 cases in blastic phase (BP).Allo-HSCT from HLA identical siblings were performed for 5 cases,HLA haplotype was performed for 14 cases and unrelated allo-HSCT for 5 cases.Twenty-four cases underwent allo-HSCT with conditioning regimen of BUCY.Prophylaxis of graft versus host disease (GVHD) included CsA + MTX plus MMF.The average follow-up was 36 months.Result All of patients were successfully engrafted.The 5-year overall survival (OS) of the 24 cases was 81％.Four patients died after allo-HSCT including 3 cases in BP from haploidentical donors and 1 case in CP from HLA identical sibling.The 5 cases who received unrelated allo-HSCT have been alive.Among the 10 cases who survived over 5 years,3 had chronic GVHD.Conclusion Children with CML could be treated effectively with allo-HSCT.There were no significant differences among different donors.Transplantation to childrcn with CML should be performed as early as possible.Preparative regimen adjustment before transplantation,the transplantation of associated comorbidities and effective prevention and treatment for CML patients after prolonged graft survival of high quality have important significance.