加强预处理方案异基因造血干细胞移植克服t(6;9)/DEK-NUP214急性髓系白血病患者不良预后

Intensive pretreatment protocol allogeneic hematopoietic stem cell transplantation to overcome poor prognosis in patients with t(6;9)/DEK-NUP214 acute myeloid leukemia

目的:探讨加强预处理方案异基因造血干细胞移植(allo-HSCT)治疗t(6;9)/DEK-NUP214急性髓系白血病(AML)的疗效。方法:对2015年3月—2023年4月应用加强预处理方案allo-HSCT治疗t(6;9)/DEK-NUP214 AML的32例患者资料进行回顾分析,观察疗效。结果:32例患者均获得造血重建,白细胞和血小板植活中位天数分别为+13(10~20)d、+12(6~29)d。预处理相关毒性(RRT)多发生于预处理2周内,Ⅰ/Ⅱ级RRT发生率为59.4%(19/32),最常见的是胃肠道反应(13/32),其次为肝损伤(9/32)。32例患者均未发生Ⅲ/Ⅳ级RRT。移植后中位随访44(3~107)个月,3年总生存(OS)率90.3%、无复发生存(RFS)率86.2%、累计复发率3.1%、移植相关死亡率9.4%。移植时完全缓解患者20例与未缓解患者12例二组比较,其3年OS率和RFS率比较差异无统计学意义(OS率:95.0%vs 82.5%,P=0.28;RFS率:87.7%vs 82.5%,P=0.61)。20例患者发病时携带FLT3-ITD与12例非携带FLT3-ITD患者比较,3年OS率和RFS率比较差异无统计学意义(OS率:95.0%vs 82.5%,P=0.3;RFS率:87.7%vs 82.5%,P=0.63)。结论:加强预处理方案allo-HSCT可克服t(6;9)/DEK-NUP214 AML患者不良预后和具有良好的耐受性,生存不受移植前状态和携带FLT3-ITD影响。

Objective To explore the efficacy of an intensified conditioning regimen allogeneic hematopoietic stem cell transplantation(allo-HSCT)for t(6;9)/DEK-NUP214 acute myeloid leukemia(AML).Methods Between 2015 and 2023,32 AML patients with t(6;9)/DEK-NUP214 fusion gene who underwent allo-HSCT were analyzed.All patients underwent an intensified conditioning regimen.Results Hematopoietic reconstitution was achieved in all 32 patients,with median days to leukocyte and platelet viability of 13(10-20)and 12(6-29)days,respectively.Pretreatment-related toxicity(RRT)occurred mostly within 2 weeks of pretreatment,with a gradeⅠ/ⅡRRT incidence of 59.3%(19/32),most commonly gastrointestinal reactions(13/32),followed by hepatic injury(9/32).No patients experienced gradeⅢ/ⅣRRT.With a median follow-up of 44(3-107)months after transplantation,the 3-year overall survival(OS)was 90.3%,relapse-free survival(RFS)was 86.2%,cumulative relapse rate was 3.1%,and transplant-related mortality was 9.4%.There were 20 patients in complete remission at the time of transplantation and 12 patients in no remission,and there was no difference in the comparison of 3-year OS and RFS between the two groups(OS:95.0%vs 82.5%,P=0.28;RFS:87.7%vs 82.5%,P=0.61).There was no difference in the 3-year OS and RFS in the 20 patients with FLT3-ITD compared with 12 patients without FLT3-ITD(OS:95.0%vs 82.5%,P=0.3;RFS:87.7%vs 82.5%,P=0.63).Conclusion Intensive pretreatment regimen allo-HSCT overcame poor prognosis and was well tolerated in patients with t(6;9)/DEK-NUP214 AML.Survival was not influenced by the state at transplant and FLT3-ITD.