Impact of minimal residual disease on the outcome of hematopoietic stem cell transplantation for childhood acute lymphoblastic leukemia within the FORUM trial.

We report the impact of MRD within the extended FORUM cohort.

我们报告了在扩展的FORUM队列中MRD的影响。

Patients (N=1,014) aged 4-21 years old, transplanted from a matched donor who had ≥1 MRD measurement prior to and/or 100 days and/or one year after HSCT were eligible.

年龄在4-21岁之间的患者（N=1,014），接受来自匹配供体的移植，供体在造血干细胞移植（HSCT）前后至少有一次微小残留病（MRD）测量，或在HSCT后100天和/或一年时进行测量，符合入选条件。

A threshold of 0.01% defined MRD positivity versus negativity.

微小残留病（MRD）阳性与阴性的界定阈值为0.01%。

Prior to HSCT, 21% of patients were MRDpos.

在造血干细胞移植（HSCT）之前，21%的患者为微小残留病灶阳性（MRDpos）。

Three-year event-free survival (EFS) was 0.73 and 0.59 (P<0.001), and 3-year cumulative incidenceof relapse (CIR) was 0.20 and 0.33 (P<0.001) in MRDneg and MRDpos patients, respectively.

三年无事件生存（EFS）率在微小残留病灶阴性（MRDneg）患者中为0.73，在MRD阳性（MRDpos）患者中为0.59（P<0.001），而三年累积复发发生率（CIR）在MRDneg和MRDpos患者中分别为0.20和0.33（P<0.001）。

The level of MRD positivity pre-HSCT (<0.1% vs. ≥0.1%) did not significantly affect outcome.

移植前微小残留病灶（MRD）阳性水平（<0.1% vs. ≥0.1%）并未显著影响结果。

Pre-HSCT MRDneg and TBI/etoposide conditioning were associated with a 2-fold lower risk of relapse, whereas MRDpos had a 2-fold higher risk of any failure and/or death.

移植前MRD阴性（MRDneg）和全身照射/依托泊苷预处理与复发风险降低2倍相关，而MRD阳性（MRDpos）则与任何失败和/或死亡风险增加2倍相关。

No detrimental effect of MRDpos pre-HSCT could be demonstrated in patients with T-cell ALL.

在异基因造血干细胞移植前的T细胞急性淋巴细胞白血病（ALL）患者中，微小残留病（MRD）阳性并未显示出有害效应。

MRDpos versus MRDneg patients at day 100 had an EFS of 0.47 versus 0.77 (P<0.001) and a CIR of 0.51 versus 0.17 (P<0.001), respectively, but post-HSCT MRDpos did not necessarily imply relapse.

在移植后第100天，微小残留病（MRD）阳性患者与MRD阴性患者的无事件生存（EFS）分别为0.47和0.77（P<0.001），累积复发率（CIR）分别为0.51和0.17（P<0.001），但异基因造血干细胞移植后MRD阳性并不一定意味着复发。

In conclusion, the MRD status pre-HSCT and at day 100 post-HSCT was a strong prognostic factor for children transplanted for ALL in the extended FORUM cohort.

总之，在扩展的FORUM队列中，接受异基因造血干细胞移植（HSCT）治疗的儿童急性淋巴细胞白血病（ALL）患者，移植前的微小残留病（MRD）状态和移植后第100天的MRD状态是强有力的预后因素。