BACKGROUND Despite the well-defined role of minimal residual disease (MRD) monitoring by real-time quantitative polymerase chain reaction (RT-PCR) for RUNX1/RUNX1T1 and CBFB-MYH11 transcripts in core binding factor (CBF) acute myeloid leukemia (AML) after intensive chemotherapy, there has been a paucity of data assessing the utility of MRD monitoring at and after allogeneic hematopoietic stem cell transplantation (HSCT). METHODS Patients with CBF AML who underwent HSCT in complete remission (first or second) from January 2007 through December 2018 were included in this analysis. RESULTS MRD by polymerase chain reaction at HSCT was assessed in 50 of 76 patients, and 44 (88%) had evidence of MRD (MRDpos). MRDpos patients had 3-year overall survival (OS) and leukemia-free survival (LFS) rates of 69.3% and 66.3%, respectively. Six MRD-negative patients had 3-year OS and LFS rates of 100% and 100%, respectively. Thirty-five of the 70 evaluable patients (50%) had a day +100 MRD assessment by RT-PCR, and 14 (40%) were MRDpos. The presence of MRD by RT-PCR on day +100 was not associated with lower estimates of LFS (75% vs 82.2%; P = .3) but was associated with a higher relapse incidence, although the difference did not reach statistical significance (27.6% vs 9.7%; P = .2). CONCLUSIONS Durable complete remissions can be achieved in patients with CBF AML with HSCT even if they are MRDpos by RT-PCR at HSCT. The clinical impact of frequent MRD monitoring for identifying a group at high risk for early relapse and then for determining the best time point for therapeutic interventions to prevent impending relapse warrants investigation in prospectively designed clinical trials.

中文翻译：

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通过实时定量聚合酶链反应监测核心结合因子急性髓性白血病对移植结局的最小残留病意义。

背景技术尽管通过实时定量聚合酶链反应（RT-PCR）对核心结合因子（CBF）急性髓细胞性白血病（AML）中的RUNX1 / RUNX1T1和CBFB-MYH11转录本进行实时最小定量残留病（MRD）监测具有明确的作用强化化疗后，缺乏数据评估异基因造血干细胞移植（HSCT）前后MRD监测的效用。方法将2007年1月至2018年12月完成HSCT完全缓解（第一次或第二次）的CBF AML患者纳入本分析。结果对76例患者中的50例进行了HSCT聚合酶链反应评估MRD，其中44例（88％）有MRD（MRDpos）证据。MRDpos患者的3年总生存率（OS）和无白血病生存率（LFS）分别为69.3％和66.3％。6名MRD阴性患者的3年OS和LFS发生率分别为100％和100％。70例可评估患者中有35例（50％）通过RT-PCR进行了每天+100 MRD评估，14例（40％）为MRDpos。在第100天时，通过RT-PCR检测到的MRD与LFS的较低估计值无关（75％比82.2％； P = 0.3），但与较高的复发率相关，尽管差异未达到统计学意义（ 27.6％和9.7％； P = 0.2）。结论CBF AML合并HSCT的患者即使通过HSCT的RT-PCR检测为MRDpos，仍可实现持久的完全缓解。频繁进行MRD监测的临床影响，用于识别早期复发的高风险人群，然后确定治疗干预措施以防止即将发生的复发的最佳时间点，需要在前瞻性设计的临床试验中进行研究。