For most patients with acute myeloid leukemia (AML), an allogeneic hematopoietic stem cell transplantation (HSCT) offers the highest chance of cure. Recently, the European LeukemiaNet (ELN) published updated recommendations on the diagnosis and risk classification in AML based on genetic factors at diagnosis as well as a dynamic adjustment (reclassification) according to the measurable residual disease (MRD) status for the favorable and intermediate risk groups. Validation of the ELN2022 risk classification has not been reported. We retrospectively analyzed 522 AML patients who received an HSCT at a median age of 59 (range 16–76) years. For patients with adequate material available and in remission prior to HSCT (n = 229), the MRD status was evaluated. Median follow-up after HSCT was 3.0 years. ELN2022 risk at diagnosis was in 22% favorable, in 26% intermediate, and in 52% adverse. ELN2022 risk at diagnosis is associated with the cumulative incidence of relapse/progression (CIR), event-free survival (EFS), and overall survival (OS) in the whole patient cohort, as well as the subgroup of patients transplanted in first remission. However, the risk stratification based on the ELN2022 classification did not significantly improve outcome prognostication in comparison to the ELN2017 classification. In our study, the newly added group of patients with myelodysplasia-related gene mutations did not have adverse outcomes. Re-classifying these patients into the intermediate risk group and adjusting the grouping for all AML patients by MRD at HSCT, led to a refined and improved risk stratification, which should be validated in independent studies.
对于大多数急性髓系白血病(AML)患者而言,异基因造血干细胞移植(HSCT)提供了最高的治愈机会。近期,欧洲白血病网(ELN)发布了基于诊断时遗传因素的AML诊断与风险分层更新建议,并对预后良好及中危组根据可测量残留病(MRD)状态进行动态调整(再分层)。目前ELN2022风险分层的验证尚未见报道。我们回顾性分析了522例接受HSCT的AML患者,中位年龄为59岁(范围16-76岁)。对于有可用样本且在移植前达到缓解的患者(n=229),我们评估了其MRD状态。移植后的中位随访时间为3.0年。根据ELN2022诊断时风险分层,22%的患者属于预后良好组,26%为中危组,52%为高危组。诊断时的ELN2022风险分层与整个患者队列以及首次缓解期移植亚组患者的累积复发/进展发生率、无事件生存期和总生存期显著相关。然而,与ELN2017分类相比,基于ELN2022的风险分层并未显著改善预后预测效果。在我们的研究中,新增的骨髓增生异常相关基因突变患者组并未出现不良结局。将这部分患者重新归类至中危组,并根据移植时的MRD状态对所有AML患者进行分组调整,形成了一种更精细且优化的风险分层方法,这需要在独立研究中进一步验证。