Introduction:Post-transplant relapse of acute myeloid leukemia and myelodysplastic syndrome faces restricted effective salvage regimens. We retrospectively analyzed the use of Azacitidine–donor lymphocyte infusion (AZA/DLI) in this setting. Furthermore, data on bone marrow Wilms tumor gene 1 (WT1) expression were collected.Methods:A Cox proportional hazards model, an outcome-oriented approach for the lowest smoothed plot of the martingale residuals, was performed for the cut-point determination of the respective WT1 expression levels. Finally, a Cox proportional hazards model investigated the association of overall survival (OS) with predictors.Results:An overall response of 41.4% with a median duration of 11.9 months for stable disease and 19.5 months for complete response (CR) patients was achieved. The disease risk index (DRI) high-/very high-risk patients had a shorter OS of 4.4 months than intermediate-risk patients, with 14.5 months,p= 0.007. At transplant, WT1-overexpressing patients (>150 copies) had a shorter median OS of 5.3 months than low-WT1-expressing ones, with 13.5 months,p= 0.024. Furthermore, patients with ≤1000 WT1 copies at relapse had a significantly longer OS with 15.3 months than patients overexpressing WT1, with 4.4 months,p= 0.0002.Conclusions:DRI and WT1 expression associate significantly with OS after AZA/DLI. Hence, WT1 may represent an MRD marker, especially in CR patients at high risk.
引言：急性髓系白血病和骨髓增生异常综合征移植后复发面临有效挽救方案有限的问题。本研究回顾性分析了阿扎胞苷联合供者淋巴细胞输注（AZA/DLI）在此类患者中的应用情况，并收集了骨髓中Wilms肿瘤基因1（WT1）表达水平的相关数据。 方法：采用Cox比例风险模型，通过基于结局的鞅残差平滑图最低点确定法，分别确定了WT1表达水平的最佳截断值。最后通过Cox比例风险模型分析总生存期（OS）与各预测因素的相关性。 结果：总体缓解率为41.4%，其中疾病稳定患者的中位缓解持续时间为11.9个月，完全缓解（CR）患者为19.5个月。疾病风险指数（DRI）高危/极高危患者的中位OS为4.4个月，显著短于中危患者的14.5个月（p=0.007）。移植时WT1高表达（>150拷贝数）患者的中位OS为5.3个月，明显短于低表达患者的13.5个月（p=0.024）。此外，复发时WT1≤1000拷贝数的患者中位OS达15.3个月，显著长于WT1高表达患者的4.4个月（p=0.0002）。 结论：DRI和WT1表达水平与AZA/DLI治疗后的OS显著相关。因此，WT1可作为微小残留病标志物，尤其适用于高危CR患者的监测。
肿瘤(癌症)患者之家