Minimally myelosuppressive regimen for remission induction in pediatric AML: long-term results of an observational study
Minimally myelosuppressive regimen for remission induction in pediatric AML: long-term results of an observational study
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儿童 AML 诱导缓解的最低限度骨髓抑制方案：观察性研究的长期结果
DOI：
10.1182/bloodadvances.2020003453
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发表时间：
2021-03-31
期刊：
影响因子：
7.5
通讯作者：
Hu, Shaoyan
中科院分区：
文献类型：
--
作者：
Hu, Yixin;Chen, Aili;Hu, Shaoyan
在资源有限的环境中，急性髓性白血病（AML）儿童的治疗失败大多数是由于拒绝治疗和毒性导致的死亡。我们最近报道了低剂量阿糖胞苷和米托蒽醌或间琥珀酸omacetaxine联合粒细胞集落刺激因子（G-CSF）（低剂量化疗[LDC]）诱导缓解，随后采用标准缓解后策略治疗AML儿童的结果。我们现在已经扩大了初始队列，并提供了长期随访。83例AML患者接受了LDC方案治疗。在研究期间，另外100名AML儿童接受了标准剂量化疗（SDC）方案。LDC组和SDC组诱导后分别有88.8%和86.4%的患者达到完全缓解（P = 0.436）。LDC组中的22名患者在第二个诱导过程中接受了SDC。显著更多的高危AML患者接受SDC方案治疗（P = 0.035）。LDC组和SDC组的5年无事件生存率无显著差异（分别为61.4% +/- 8.7% vs 65.2% +/-7.4%; P = 0.462），总生存期（分别为72.7% ± 6.9%和72.5% ± 6.2%; P = 0.933）和复发率（分别为20.5% ± 4.5%和17.6% ± 3.9%; P = 0.484）。在LDC和SDC组中，基于完全缓解时平均变异等位基因频率的突变清除率在诱导I后为1.9% vs 0.6%（P <0.001），在诱导II后为0.17% vs 0.078%（P = 0.052）。总之，我们的研究证实了至少接受1个疗程LDC治疗的AML儿童的高缓解率。这些结果虽然是初步的，但也表明这些儿童的长期生存率与接受SDC方案的儿童相当。
Treatment refusal and death as a result of toxicity account for most treatment failures among children with acute myeloid leukemia (AML) in resource-constrained settings. We recently reported the results of treating children with AML with a combination of low-dose cytarabine and mitoxantrone or omacetaxine mepesuccinate with concurrent granulocyte colony-stimulating factor (G-CSF) (low-dose chemotherapy [LDC]) for remission induction followed by standard postremission strategies. We have now expanded the initial cohort and have provided long-term follow-up. Eighty-three patients with AML were treated with the LDC regimen. During the study period, another 100 children with AML received a standard-dose chemotherapy (SDC) regimen. Complete remission was attained in 88.8% and 86.4% of patients after induction in the LDC and SDC groups, respectively (P = .436). Twenty-two patients in the LDC group received SDC for the second induction course. Significantly more high-risk AML patients were treated with the SDC regimen (P = .035). There were no significant differences between the LDC and SDC groups in 5-year event-free survival (61.4% +/- 8.7% vs 65.2% +/- 7.4%, respectively; P = .462), overall survival (72.7% +/- 6.9% vs 72.5% +/- 6.2%, respectively; P = .933), and incidence of relapse (20.5% +/- 4.5% vs 17.6% +/- 3.9%, respectively; P = .484). Clearance of mutations based on the average variant allele frequency at complete remission in the LDC and SDC groups was 1.9% vs 0.6% (P < .001) after induction I and 0.17% vs 0.078% (P = .052) after induction II. In conclusion, our study corroborated the high remission rate reported for children with AML who received at least 1 course of LDC. The results, although preliminary, also suggest that longterm survival of these children is comparable to that of children who receive SDC regimens.