Objective: To evaluate the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for the treatment of acute myeloid leukemia (AML) patients with FLT3-ITD mutation. Methods: From September 2008 to December 2016, 40 AML patients with FLT3-ITD mutation were enrolled in the study. The therapeutic process, outcomes and prognostic factors were retrospectively analyzed. Results: The median of WBC at initial diagnosis was 35.0 (range 1.7-185.0) ×10(9)/L. The median course number of chemotherapy was 4 (range 2-7). At the time of transplantation, 34 patients were at the first complete remission (CR(1)) stage, and the other 6 ones were non-remission after chemotherapy. 24 patients received allogeneic transplants from an HLA-matched sibling donor, 7 cases from a HLA-matched unrelated donor, the remaining 9 ones received allograft from a haploidentical donor. The rate of 3-year overall survival (OS) and disease free survival (DFS) in all patients were both 74.3% (95% CI 60.4%-88.2%). The 3-year cumulative incidences of disease relapse and non-relapse mortality were 7.5% (95%CI 1.9%-18.4%) and 18.2% (95% CI 7.9%-32.0%), respectively. More than one course of chemotherapy before achieving CR(1) and the occurrence of acute GVHD after transplantation were associated with poor outcome in terms of OS and DFS. The relapse rates were significantly lower in patients receiving transplantation at CR(1) stage [0 vs 50.0% (95%CI 77.7%-82.9%) , P<0.001] and achieving CR(1) after one course induction therapy [0 vs 16.7% (95%CI 3.9%-37.3%) , P=0.020]. Conclusions: Allo-HSCT was an efficient approach for AML patients with FLT3-ITD mutation. Patients obtained better survival, especially for those achieving CR after one course induction therapy and receiving transplantation at CR(1) stage.
目的： 总结异基因造血干细胞移植（allo-HSCT）治疗成人FLT3-ITD阳性急性髓系白血病（AML）的远期疗效。 方法： 回顾性分析2008年9月至2016年12月于中国医学科学院血液病医院行allo-HSCT的40例FLT3-ITD阳性AML（M(3)除外）患者临床资料，对移植疗效及预后相关因素进行统计学分析。 结果： 40例FLT3-ITD阳性AML患者，男16例，女24例，中位年龄为39.5（16~54）岁，初诊时WBC中位数为35.0（1.7~185.0）×10(9)/L，移植前化疗中位疗程数为4（2~7）个。移植前第1次完全缓解（CR(1)）34例，未缓解（NR）6例。同胞全相合移植24例，HLA配型相合无关供者移植7例，单倍型移植9例。患者3年总生存（OS）率为74.3%（95%CI 60.4%~88.2%），无病生存（DFS）率为74.3%（95%CI 60.4%~88.2%）。3年累积复发率为7.5%（95%CI 1.9%~18.4%），累积移植相关死亡率（TRM）为18.2%（95%CI 7.9%~32.0%）。多因素分析结果显示，1个疗程未达CR（HR=4.388，95% CI 1.232~15.637，P=0.023）、发生急性GVHD（HR=5.721，95% CI 1.454~22.518，P=0.013）是影响患者OS的独立预后危险因素。CR(1)期行allo-HSCT[0对50.0%（95%CI 77.7%~82.9%），P<0.001]及1个疗程达CR [0对16.7%（95%CI 3.9%~37.3%），P=0.020]患者3年累积复发率分别较NR及1个疗程未达CR患者显著下降。 结论： allo-HSCT可以改善FLT3-ITD突变阳性AML患者预后，诱导化疗期间1个疗程达CR及CR(1)期行allo-HSCT患者预后相对较好。.
Keywords: Gene, FLT3; Hematopoietic stem cell transplantation; Leukemia, myeloid, acute.