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

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年5月2日

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  • Ruoxuan Zhang,
  • Huihong Huang,
  • Yi Zhang,
  • Yi Xia,
  • Jiayu Huang,
  • Chuanhe Jiang,
  • Luxiang Wang,
  • Haiyang Lu,
  • Zengkai Pan,
  • Gaoxiang Wang,
  • Yang Yang,
  • Yilei Ma,
  • Xiaodong Mo,
  • Wei Shi,
  • Xiaoxia Hu,
  • Yang Cao

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.