文章：

伴KMT2A（MLL）重排的急性髓系白血病的结局：TROPHY研究组的多中心研究

Outcomes of acute myeloid leukemia with KMT2A (MLL) rearrangement: a multicenter study of TROPHY group

原文发布日期：2025-05-02

DOI: 10.1038/s41408-025-01293-x

类型: Article

开放获取: 是

英文摘要：

Acute myeloid leukemia (AML) with KMT2A rearrangement (KMT2A-r) is associated with poor prognosis, but the benefit of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for KMT2A-r AML is unclear. We reviewed adult AML patients treated within the TROPHY group and identified 292 cases of KMT2A-r AML, 254 (87.0%) of whom achieved first complete remission (CR1) and 192 (75.6%) of CR1 patients underwent allo-HSCT. We show that allo-HSCT is an independent favorable prognostic factor in CR1 patients for both overall survival (OS) (hazard ratio [HR] = 0.56, 95% confidence interval [CI]: 0.45–0.69, P < 0.001) and cumulative incidence of relapse (CIR) (HR = 0.01, 95% CI: 0.005–0.04, P < 0.001). Among allo-HSCT recipients, survival outcomes were comparable between patients with KMT2A::MLLT3 and those with other 11q23/KMT2A rearrangements (3-year OS: 74.3% vs. 77.5%, P = 0.97; 3-year event-free survival [EFS]: 55.2% vs. 62.2%, P = 0.34; 3-year CIR: 24.4% vs. 20.8%, P = 0.32). Both multiparameter flow cytometry-based measurable residual disease (MFC-MRD) and KMT2A-r MRD determined by quantitative PCR prior to allo-HSCT were associated with worse transplant outcomes. Multivariable analysis identified detectable KMT2A-r MRD at allo-HSCT as a significant risk factor for reduced EFS (HR = 2.46, 95% CI: 1.32–4.60, P = 0.005). These findings confirm the survival benefit of allo-HSCT in adult patients with KMT2A-r AML and underscore the prognostic value of KMT2A-r MRD prior to transplantation.
 

摘要翻译： 

伴有KMT2A重排（KMT2A-r）的急性髓系白血病（AML）预后较差，但异基因造血干细胞移植（allo-HSCT）对KMT2A-r AML的益处尚不明确。我们回顾了TROPHY研究组治疗的成人AML患者，共纳入292例KMT2A-r AML病例，其中254例（87.0%）获得首次完全缓解（CR1），而CR1患者中有192例（75.6%）接受了allo-HSCT。研究显示，在CR1患者中，allo-HSCT是总生存期（OS）（风险比[HR]＝0.56，95%置信区间[CI]：0.45–0.69，P＜0.001）和累积复发率（CIR）（HR＝0.01，95% CI：0.005–0.04，P＜0.001）的独立有利预后因素。在接受allo-HSCT的患者中，KMT2A::MLLT3与其他11q23/KMT2A重排亚型的生存结局相当（3年OS：74.3%对比77.5%，P＝0.97；3年无事件生存期[EFS]：55.2%对比62.2%，P＝0.34；3年CIR：24.4%对比20.8%，P＝0.32）。无论基于多参数流式细胞术的可测量残留病（MFC-MRD）还是移植前定量PCR检测的KMT2A-r MRD，均与较差的移植结局相关。多变量分析确定移植时可检测的KMT2A-r MRD是EFS降低的显著风险因素（HR＝2.46，95% CI：1.32–4.60，P＝0.005）。这些结果证实了allo-HSCT在成人KMT2A-r AML患者中的生存获益，并强调了移植前KMT2A-r MRD检测的预后价值。

原文链接：

Outcomes of acute myeloid leukemia with KMT2A (MLL) rearrangement: a multicenter study of TROPHY group