Low- vs standard-dose regimens as induction for pediatric AML: a multicenter, randomized noninferiority trial.

We aimed to compare the efficacy and safety of a low-dose chemotherapy (LDC) regimen for induction of AML.

我们旨在比较低剂量化疗（LDC）方案诱导AML的疗效和安全性。

A randomized, multicenter, noninferiority trial was conducted in patients with AML aged <18 years.

进行了一项随机、多中心、非劣效试验，对象为年龄小于18岁的急性髓细胞性白血病（AML）患者。

Patients received low-dose cytarabine, mitoxantrone or idarubicin, and granulocyte colony-stimulating factor (G-CSF) or standard-dose chemotherapy (SDC; cytarabine, daunomycin, and etoposide).

患者接受低剂量阿糖胞苷、米托蒽醌或伊达比星，以及粒细胞集落刺激因子（G-CSF）或标准剂量化疗（SDC；阿糖胞苷、柔红霉素和依托泊苷）。

All patients received postremission consolidation with standard chemotherapy and/or hematopoietic stem cell transplantation.

所有患者接受了标准化疗和/或造血干细胞移植的缓解后巩固治疗。

The primary end point was to compare response rates between treatments.

主要终点是比较不同治疗方案的反应率。

The secondary end points were to compare the outcomes, toxicity, and safety of the LDC and SDC regimens.

次要终点是比较LDC和SDC方案的疗效、毒性和安全性。

The 2 treatment arms showed no significant differences in outcomes.

两个治疗组在疗效上没有显著差异。

Complete remission (CR)/CR with incomplete count recovery rates after induction were 95.1% and 95.3% in the LDC and SDC arms, respectively.

诱导治疗后完全缓解(CR)/CR伴不完全血细胞恢复率在LDC和SDC组分别为95.1%和95.3%。

Measurable residual disease <0.1% after induction II was observed in 87.4% and 87.1% of patients in the LDC and SDC arms, respectively.

诱导治疗II后，LDC和SDC组分别有87.4%和87.1%的患者可测量的残余疾病<0.1%。

Median time to neutrophil and platelet recovery was significantly shorter among patients receiving the LDC regimen.

接受LDC方案治疗的患者中性粒细胞和血小板恢复的中位时间显著缩短。

Patients in the LDC arm had a 4-year overall survival(OS) of 81.3% vs 83.6% (P = .611), and a 4-year event-free survival (EFS) of 61.5% vs 63.1% (P = .832).

LDC组患者的4年总生存（OS）为81.3%，对照组为83.6%（P = .611），4年无事件生存（EFS）为61.5%，对照组为63.1%（P = .832）。

In conclusion, the LDC regimen was well tolerated, and was associated with CR, EFS, and OS rates that were not inferior to those of patients treated with the SDC regimen.

总之，LDC方案耐受性良好，与SDC方案治疗的患者相比，其完全缓解（CR）、无事件生存（EFS）和总生存（OS）率并无劣势。

The trial was registered at www.chictr.org.cn as ChiCTR1800015883.

该试验已在www.chictr.org.cn上注册，注册号为ChiCTR1800015883。