异基因造血干细胞移植治疗Fms样酪氨酸激酶3基因内部串联重复(FLT3-ITD)阳性的急性髓性白血病的疗效分析

 目的 分析异基因造血干细胞移植(allo-HSCT)治疗伴有Fms样酪氨酸激酶3基因内部串联重复(FLT3-ITD)阳性的急性髓性白血病(AML)的疗效,探讨不同移植方式及疾病状态对该类患者预后的影响。方法 2006年10月至2012年10月在苏州大学附属第一医院行allo-HSCT的AML患者共314例,其中FLT3-ITD阳性54例,回顾性分析allo-HSCT对FLT3-ITD阳性AML患者的临床疗效。结果 54例FLT3-ITD阳性患者3年总生存(OS)率为56%, 3年无白血病生存(LFS)率为47%。其中同胞人类白细胞抗原(HLA)全相合及亲缘HLA单倍型相合造血干细胞移植的患者3年OS率分别为56%和60%,3年LFS率为45%和54%。两组在OS时间及LFS时间方面的差异均无统计学意义(χ²=0.074,P=0.786; χ²=0.006,P=0.941)。47例(87.0%)患者移植前本病处于首次完全缓解(CR1),7例(13.0%)患者移植前本病处于非CR1期。处于CR1期的患者的生存显著优于非CR1期患者。结论 allo-HSCT是FLT3-ITD阳性AML患者的有效治疗方法,亲缘HLA单倍型相合造血干细胞移植与同胞HLA全相合移植疗效相似。疾病复发是影响其疗效的主要因素。
   

Clinical outcome of FLT3-ITD (+) acute myeloid leukemia patients treated with allogeneic hematopoietic stem cell transplantation

Objective To study the clinical outcome of patients with fms-like tyrosine kinase-3 internal tandem duplication(FLT3-ITD) positive acute myeloid leukemia (AML) treated with allogeneic hematopoietic stem cell transplantation (allo-HSCT) and to explore the potential prognostic factors to patients′ survival including transplant types or disease status.Methods A total of 314 AML patients in our center from October 2006 to October 2012 were retrospectively analyzed, among whom, 54 patients were defined with FLT3-ITD positive. Survival rates and treatment-related mortality were further analyzed.ResultsFor all 54 FLT3-ITD positive patients, the 3-year overall survival (3-OS) rate was 56% and 3-year leukemia-free survival (3-LFS) rate was 47%. The outcome of haplo-identical HSCT was similar as that of sibling donors(3-OS rate: 60% vs 54%; 3-LFS rate: 54% vs 45%, respectively). There were 47 patients who received transplantation in first complete remission(CR1). The other 7 patients were of disease relapse or in CR2 before transplantation. Not surprisingly, patients in CR1 had better prognosis than those in non-CR1.Conclusions Allo-HSCT is an effective treatment for AML patients with FLT3-ITD positive mutation. The survival outcome of haplo-identical HSCT was comparable with that of sibling donors. Relapse of AML was the dominant factor related to the mortality of FLT3-ITD positive AML patients after allo-HSCT.