Kudo Kazuko, Kojima Seiji, Tabuchi Ken, Yabe Hiromasa, Tawa Akio, Imaizumi Masue, Hanada Ryoji, Hamamoto Kazuko, Kobayashi Ryoji, Morimoto Akira, Nakayama Hideki, Tsuchida Masahiro, Horibe Keizo, Kigasawa Hisato, Tsukimoto Ichiro
Department of Pediatrics, Institute of Clinical Medicine, University of Tsukuba, Tsukuba, Japan.
J Clin Oncol. 2007 Dec 1;25(34):5442-7. doi: 10.1200/JCO.2007.12.3687.
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.
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.
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).
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.
评估一种专门为唐氏综合征（DS）合并急性髓系白血病（AML）患者设计的强度较低的化疗方案，并确定无事件生存的预后因素。
72例AML-DS患者接受缓解诱导化疗，方案包括吡柔比星（25mg/m²/d，共2天）、阿糖胞苷（100mg/m²/d，共7天）和依托泊苷（150mg/m²/d，共3天）。患者接受四个疗程的相同方案强化治疗。未包括中枢神经系统白血病的预防措施。
除两名患者外，所有患者年龄均小于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（EFS）率为83%±9%。9例患者复发，1例在CR期间因肺炎死亡。多因素分析显示，7号染色体单体的存在是不良预后的更大危险因素（比值比=5.67；P=.027）。
强度较低的化疗方案在标准风险AML-DS患者中产生了优异的疗效。未来AML-DS试验应考虑以风险为导向的治疗。
