异基因造血干细胞移植治疗儿童急性髓系白血病的临床观察

Study of allogeneic stem cell transplantation for pediatric acute myeloid leukemia

目的总结分析异基因造血干细胞移植(allogeneic hematopoietic stem cell transplantation,allo-HSCT)治疗儿童急性髓系白血病(acute myeloid leukemia,AML)的临床疗效,发现可能影响预后的相关因素。方法收集2006年1月至2016年1月首都医科大学附属北京儿童医院血液肿瘤中心确诊为AML并进行allo-HSCT治疗的病例,采用Kaplan-Meier生存曲线评估患者的总生存率(overall survival,OS),采用Log-rank检验进行不同组别比较。多因素分析采用Cox回归法分析影响预后的相关因素。结果共收集46例病例,中位随访时间27个月。存活31例,15例死亡,8例复发。5年OS为(61. 1±8. 7)%,中位生存时间为77. 3个月,5年累积复发率为(16. 0±6. 0)%。Log-rank检验结果显示前期后期不同移植方案、移植前缓解状态、缓解次数、不同供者来源、是否合并移植物抗宿主病在对OS影响上差异无统计学意义(P>0. 05)。多因素分析显示,合并髓系肉瘤和复发是影响患者预后的危险因素(P <0. 05)。结论异基因造血干细胞移植是治疗AML尤其是难治/复发AML的有效治疗手段,随着移植方案的改良及支持治疗的发展,生存率逐年提高。合并髓系肉瘤及复发是影响AML移植治疗预后的重要不良因素。

Objective To analysis the effectiveness of allogeneic hematopoietic stem cell transplantation(allo-HSCT)for treatment of children with acute myeloid leukemia(AML),and to identify the possible prognostic factors for treatment.Methods We retrospectively reviewed outcomes of children with AML who underwent allo-HSCT at Hematology Oncology Center of Beijing Children’s Hospital,Capital Medical University between January of 2006 and January of 2016.The overall survival(OS)were estimated by Kaplan-Meier method,Log-rank test was used to compare the different groups and prognostic factors were evaluated by Cox regression.Results A total of 46 patients were involved,with 31 patients survived,and 15 patients died,8 patients relapsed,with an average follow-up time of 27 months.5 years OS was(61.1±8.7)%,median survival time was 77.3 months,and 5 years cumulative relapse rate was(16.0±6.0)%.The results of Log-rank test indicated no significant difference(P>0.05)with different protocol,remission state,remission times,graft versus-host disease(GVHD)and donors.The Cox regression suggested myeloid sarcoma and the relapse are the prognostic factors(P<0.05).Conclusion Allogeneic hematopoietic stem cell transplantation is an effective treatment for AML,especially,refractory/relapsed AML.With the modified conditioning regimen and supportive treatment,the survival rate is increasing year by year.Myeloid sarcoma and relapse are important adverse prognostic factors of HSCT treatment for AML.