【摘要】 目的：分析移植前和移植后早期骨髓WT1表达水平对在首次完全缓解期（CR1）行异基因造血干细胞移植（allo-HSCT）的急性髓性白血病（AML）患者预后的影响。方法：回顾性分析2012年5月至2021年12月间在本中心于CR1期行allo-HSCT的107例成人AML患者的临床资料，结合临床相关因素探讨了移植前、移植后3个月和移植的6个月骨髓WT1表达水平对患者复发及生存的影响。结果：107例患者的中位随访时间为70(11-117)个月，其中15例患者死亡。Kaplan-Meier生存分析显示，3年总体生存（OS）率为85.0%。20例患者出现复发，复发的中位时间为8(0.5-44)个月，1年累积复发率为13.1%。分析患者移植前、移植后3个月和移植后6个月的WT1检测数据，WT1总体中位值为0.26%(0%-23.64%)，上四分位值为0.74%，且移植前、移植后3个月和移植后6个月的WT1表达水平无显著统计学差异（P=0.227）。单因素分析结果显示，与移植后3个月WT1水平≤0.74的患者相比，移植后3个月WT1水平>0.74%的患者一年复发率明显升高（P=0.029）,3年OS率明显降低（P<0.001）。其他影响复发的因素为干细胞来源（P=0.041）和cGVHD(P=0.013)；影响3年OS的其他因素包括遗传高危（P=0.048）和干细胞来源（P=0.016）。多因素分析结果显示，移植后3个月WT1水平>0.74%有影响一年复发率的趋势（HR=3.309,95%CI:0.958-11.431,P=0.058），而未发生cGVHD是影响1年复发的独立危险因素（HR=3.473,95%CI:0.749-16.100,P=0.037）；而移植后3个月WT1水平>0.74%为患者移植后3年OS的独立危险因素（HR=6.886,95%CI:2.402-19.738,P<0.001）。结论：CR1期行allo-HSCT的AML患者移植后3个月WT1高表达影响患者1年复发率和3年OS，且是影响3年OS的独立危险因素，提示动态监测WT1表达水平在CR1期行allo-HSCT的AML患者预后评估中有一定价值。更多还原

【Abstract】 Objective: To analyze the predictive role of WT1 expression levels pre-and early post-transplantation on relapse and overall survival(OS) in patients with acute myeloid leukemia(AML) undergoing allogeneic hematopoietic stem cell transplantation(allo-HSCT) during their first complete remission(CR1). Methods: A retrospective analysis was conducted on the clinical data of 107 adult AML patients who underwent allo-HSCT during their CR1 at our center between May 2012 and December 2021. The predictive role of bone marrow WT1 expression levels before transplantation and at 3 and 6 months post-transplantation on relapse and OS was explored in combination with relevant clinical factors. Results: The median follow-up time for the 107 patients was 70(range: 11-117) months. Among the patients, 15 cases died. Kaplan-Meier survial analysis showed that the 3-year overall survival(OS) rate was 85.0%. 20 patients experienced relapse, with a median time to relapse of 8(range: 0.5-44) months and a 1-year cumulative relapse rate of 13.1%. The overall median value of WT1 before transplantation, 3 months after transplantation, and 6 months after transplantation was 0.26%(range: 0%-23.64%), with an upper quartile value of 0.74%. No statistically significant differences in WT1 expression levels were observed among the pre-transplantation, 3-month post-transplantation, and 6-month posttransplantation time points(P=0.227). Univariate analysis showed that patients with WT1 levels >0.74% at 3 months post-transplantation had a higher 1-year relapse rate(P=0.029) and lower 3-year OS rate(P<0.001) compared to patients with WT1 levels ≤0.74%. Other significant factors affecting 1-year relapse included stem cell source(P=0.041) and chronic graft-versus-host disease(cGVHD)(P=0.013). For 3-year OS, additional influencing factors were genetic high risk(P=0.048) and stem cell source(P=0.016). Multivariate analysis revealed that WT1 level >0.74% at 3 months posttransplantation had a trend to affect 1-year relapse rate(HR=3.309, 95%CI: 0.958-11.431, P=0.058), while the absence of cGVHD was an independent risk factor for 1-year relapse(HR=3.473, 95%CI: 0.749-16.100, P=0.037). Only WT1 level >0.74% at 3 months post-transplantation was an independent risk factor for 3-year OS(HR=6.886, 95%CI: 2.402-19.738, P<0.001). Conclusion: High WT1 expression level at 3 months post-transplantation in AML patients undergoing alloHSCT during CR1 affects the 1-year relapse rate and 3-year OS, and is an independent risk factor affecting 3-year OS. These findings suggest that dynamic monitoring of WT1 expression levels has certain value in prognostic assessment of AML patients who received allo-HSCT during CR1.更多还原