单倍体相合造血干细胞移植治疗中危NPM1wt/non-CEBPAdm/FLT3-ITD^neg急性髓系白血病的疗效分析

Efficacy of Haploidentical Hematopoietic Stem Cell Transplantation in Treatment of Intermediate Risk Acute Myeloid Leukemia with Negative for FLT3-ITD,NPM1 or Biallelic CEBPA Mutation

目的:探讨三阴性(NPM1wt/non-CEBPAdm/FLT3-ITD^neg)中危急性髓系白血病患者(acute myeloid leukemia, AML)诱导化疗达首次缓解(complet remission,CR1)后不同治疗方式的疗效。方法:回顾性分析了2009年10月至2016年5月苏州大学附属第一医院血液科收治的268名中危NPM1wt/non-CEBPAdm/FLT3-ITD^neg急性髓系白血病患者的临床资料,对移植疗效及预后相关因素进行统计学分析。结果:化疗组、同胞全相合造血干细胞移植组及单倍体相合造血干细胞移植组的2年总体生存(OS)率分别为63.7%、71.7%和75.5%(P<0.05);2年无复发生存(RFS)率分别为52.8%、67.1%和71. 3%(P<0.001);累积复发率(CIR)分别为24.7%、16.9%和14.4%(P<0.05);非复发死亡率(NRM)分别为26.2%、17.3%、14.4%(P>0.05)。移植相关不良事件分析显示,MSD-HSCT组与haploidentical-HSCT组Ⅱ-Ⅳ度aGVHD分别为48.9%和45.6%(P>0.05);广泛c GVHD分别为21.6%和8.8%(P<0.05)。结论:中危NPM1wt/non-CEBPAdm/FLT3-ITD^neg急性髓系白血病患者首次缓解后行同胞全相合或单倍体相合造血干细胞移植的效果优于单纯巩固化疗。单倍体相合造血干细胞移植与同胞全相合造血干细胞移植在巩固治疗效果和移植相关死亡率上均无明显差异。

Objective: To compare the efficacy of haploidentical hematopoietic stem cell transplantation(hi-HSCT) HLA-matched sibling donor hematopoietic stem cell transplantation(MSD-HSCT) and post-remission chemotherapy(PR-CT) in treatment of intermediate risk acute myeloid leukemia with negative for FLT3-ITD, NPM1 or biallelic CEBPA mutation. Methods: The clinical data of patients with intermediate risk NPM1wt/non-CEBPAdm/FLT3-ITDneg AML from October 2009 to May 2016 were retrospectively analyzed. Results: The overall survival rate of the patients treated with PR-CT, MSD-HSCT or hi-HSCT was 63.7%, 71.7%, 75.5%, respectively(P<0.05);the disease-free survival(DFS) rate was 52.8%, 67.1%, 71.3% respectively(P<0.001);the cumulative incidence of relapse was 24.7%, 16.9%, 14.4% respectively(P<0.05);the non-relapse mortality was 26.2%, 17.3%, 14.4% reapectively(P>0.05). The analysis of transplantation, related adverse events showed that Ⅱ-Ⅳ grade of aGVHD in the MSD-HSCT group and hiHSCT group was 48.9% and 45.6% respectively(P>0.05);the extensive cGVHD event was 21.6% and 8.8%(P<0.05) respectively. Conclusion: The efficiency of hi-HSCT and MSD-HSCT is superior to that of PR-CT for treatment of patients with intermediate risk NPM1wt/non-CEBPAdm/FLT3-ITDneg AML after CR1, there is no statistically significant difference in the efficiency of consolidatorg treatment and the transplantation-related mortality between hi-HSCT and MSD-HSCT.