文章：

儿童急性髓系白血病诱导化疗：双药与三药方案的随机对照试验

Two-drug versus three-drug induction chemotherapy in pediatric acute myeloid leukemia: a randomized controlled trial

原文发布日期：2022-09-06

DOI: 10.1038/s41408-022-00726-1

类型: Article

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英文摘要：

The benefit of three-drug induction chemotherapy over a two-drug induction has not been evaluated in pediatric acute myeloid leukemia (AML). We, therefore, conducted a randomized controlled trial to ascertain the benefit of a three-drug induction regimen. Patients aged 1–18 years with newly diagnosed AML were randomized to two cycles of induction chemotherapy with daunorubicin and ara-C (DA) or two cycles of ara-C, daunorubicin, and etoposide (ADE). After induction, patients in both arms received consolidation with two cycles of high-dose ara-C. The study’s primary objective was to compare the event-free survival (EFS) between the two arms. The secondary objectives included comparing the composite complete remission (cCR) rates, overall survival (OS), and toxicities. The study randomized 149 patients, 77 in the DA and 72 in the ADE arm. The median age was 8.7 years, and 92 (62%) patients were males. The median follow-up was 50.9 months. The cCR rate in the DA and ADE arm were 82% and 79% (p = 0.68) after the second induction. There were 13 (17%) induction deaths in the DA arm and 12 (17%) in the ADE arm (p = 0.97). The 5-year EFS in the DA and ADE arm was 34.4% and 34.5%, respectively (p = 0.66). The 5-year OS in the DA and ADE arms was 41.4% and 42.09%, respectively (p = 0.74). There were no significant differences in toxicities between the regimens. There was no statistically significant difference in EFS, OS, CR, or toxicity between ADE and DA regimens in pediatric AML. The trial was registered with the Clinical Trial Registry of India (Reference number: CTRI/2014/11/005202).
 

摘要翻译： 

在儿童急性髓系白血病（AML）中，三药诱导化疗相较于两药诱导的益处尚未得到评估。为此，我们开展了一项随机对照试验，以明确三药诱导方案的获益情况。年龄在1-18岁、新诊断为AML的患者被随机分配接受两个周期的柔红霉素和阿糖胞苷（DA）诱导化疗，或两个周期的阿糖胞苷、柔红霉素和依托泊苷（ADE）诱导化疗。诱导治疗后，两组患者均接受两个周期大剂量阿糖胞苷的巩固治疗。本研究的主要目的是比较两组患者的无事件生存率（EFS）。次要目标包括比较复合完全缓解率（cCR）、总生存率（OS）以及毒性反应。研究共随机纳入149例患者，其中DA组77例，ADE组72例。中位年龄为8.7岁，92例（62%）为男性。中位随访时间为50.9个月。第二次诱导后，DA组和ADE组的cCR率分别为82%和79%（p=0.68）。DA组有13例（17%）诱导期死亡，ADE组有12例（17%）（p=0.97）。DA组和ADE组的5年EFS分别为34.4%和34.5%（p=0.66），5年OS分别为41.4%和42.09%（p=0.74）。两种方案的毒性反应无显著差异。在儿童AML中，ADE方案与DA方案在EFS、OS、CR或毒性方面均无统计学显著差异。该试验已在印度临床试验注册中心注册（注册号：CTRI/2014/11/005202）。

原文链接：

Two-drug versus three-drug induction chemotherapy in pediatric acute myeloid leukemia: a randomized controlled trial