Prospective study of a pirarubicin, intermediate-dose cytarabine, and etoposide regimen in children with down syndrome and acute myeloid leukemia: The Japanese childhood AML cooperative study group
Prospective study of a pirarubicin, intermediate-dose cytarabine, and etoposide regimen in children with down syndrome and acute myeloid leukemia: The Japanese childhood AML cooperative study group
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DOI：
10.1200/jco.2007.12.3687
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发表时间：
2007-12-01
影响因子：
45.3
通讯作者：
Tsukimoto, Ichiro
中科院分区：
文献类型：
--
作者：
Kudo, Kazuko;Kojima, Seiji;Tsukimoto, Ichiro
目的探讨唐氏综合征（Down syndrome，DS）合并急性髓细胞白血病（acute myeloid leukemia，AML）患者的低强度化疗方案及无事件生存率的影响因素。（25 mg/m2/d，持续2天）、阿糖胞苷（100 mg/m2/d，持续7天）和依托泊苷（150 mg/m2/d，持续3天）。患者接受相同方案的4个疗程强化治疗。结果除2例患者外，所有患者均小于4岁，72例患者中有67例（93%）被诊断为急性巨核细胞白血病（AMKL）。72例患者中有70例（97.2%）达到完全缓解（CR），估计4年无事件生存率（EFS）为83% ± 9%。9名患者复发，1名患者在CR期间因肺炎死亡。多因素分析显示，7号单体的存在是不良结局的更大危险因素（OR = 5.67; P = 0.027）。在未来的AML-DS试验中应考虑风险导向治疗。
Purpose To evaluate a less intensive chemotherapeutic regimen specifically designed for patients with Down syndrome (DS) and acute myeloid leukemia (AML), and to determine the prognostic factors for event-free survival.Patients and Methods Seventy-two patients with AML-DS were treated with remission induction chemotherapy consisting of pirarubicin (25 mg/m2/d for 2 days), cytarabine (100 mg/m2/d for 7 days), and etoposide (150 mg/m2/d for 3 days). Patients received four courses of intensification therapy of the same regimen. Prophylaxis for CNS leukemia was not included.Results All but two patients were younger than 4 years, and 67 of the 72 patients (93%) were diagnosed as acute megakaryoblastic leukemia (AMKL). Seventy of the 72 patients (97.2%) achieved a complete remission (CR), and the estimated 4-year event-free survival (EFS) rate was 83% +/- 9%. Nine patients relapsed, and one died as a result of pneumonia during CR. Multivariate analysis revealed that the presence of monosomy 7 was a greater risk factor of adverse outcome (odds ratio = 5.67; P =.027).Conclusion A less intensive chemotherapeutic regimen produces excellent outcomes in standard-risk AML-DS patient. Risk-oriented therapy should be considered for future trials in AML-DS.