Objective: To compare the efficacy and toxicity of 10 mg/m² or 8 mg/m² idarubicin (Ida) combined with cytarabine (IA"3+7"regimen) as induction chemotherapy for adult patients with newly diagnosed acute myeloid leukemia (AML).
Methods: From June 2004 to October 2013, 335 adult AML (non acute promyelocytic leukemia) patients receiving the IA regimen as induction chemotherapy were enrolled, including 198 cases with 10 mg/m² Ida and 137 cases with 8 mg/m² Ida for 3 days. We compared the hematologic response, hematologic side effects and prognosis between the two regimens.
Results: Except for 4 early deaths, the complete remission (CR) rate after the first cycle of induction chemotherapy was 72.5%, 10.0% partial remission (PR) and 82.5% overall remission (OR) rate. The CR and OR rates were higher in the 10 mg/m² Ida group than the 8 mg/m² Ida group (CR: 78.9% vs 63.5%, P=0.003; OR: 88.2% vs 75.4%, P=0.007). Multivariate analysis showed that female, HGB≥100 g/L, FLT3-ITD mutation negative and 10 mg/m² Ida were favorable factors for CR. All patients presented cytopenias of grade Ⅳ. There was no differences on the recovery time of ANC≥0.5×10⁹/L and PLT≥20×10⁹/L after induction chemotherapy. Within a median follow-up of 14 (1-118) months, 98 (29.3%) patients relapsed, 92 (27.5%) died. The disease-free survival (DFS) and overall survival (OS) at 3 years were 53.2% and 58.9%, respectively. DFS and OS at 3-year were 34.2% and 37.4% in the chemotherapy cohort, 74.5% and 81.2% in the transplant cohort. 10 mg/m² Ida was an independent favorite factor for DFS (P=0.040) and OS (P=0.007).
Conclusion: As compared to 8 mg/m², 10 mg/m² Ida significantly improved the CR, with the same extent of hematological side effects, and was an independent favorite factor for DFS and OS.
目的: 比较去甲氧柔红霉素（IDA）10 mg/m2和8mg/m2（简称10和8 mg组）联合阿糖胞苷（IA）方案诱导治疗成人初发急性髓系白血病（AML）（除外急性早幼粒细胞白血病）患者的疗效、血液学不良反应及预后。
方法: 回顾性分析2004年6月至2013年10月335例成人初发AML患者资料，其中IDA 10 mg组198例，8 mg组137例，比较两组患者的第1疗程化疗反应、血液学不良反应及早期死亡率，分析IDA剂量对预后的影响。
结果: 除外早期死亡的4例患者，331例可评估患者的第1疗程完全缓解（CR）率为72.5%，部分缓解（PR）率为10.0%，总反应（OR）率为82.5%。IDA 10 mg组第1疗程CR率及OR率均显著高于IDA 8 mg组（CR率：78.9%对63.5%，P=0.003；OR率：88.2%对75.4%，P=0.007）。多因素分析显示，女性、初诊时HGB≥100 g/L、FLT3-ITD突变阴性、IDA剂量10 mg/m2是有利于第1疗程获得CR的因素。化疗后所有患者均出现4级血细胞减少，两组患者化疗后中性粒细胞绝对值≥0.5×109/L和PLT≥20×109/L恢复时间差异无统计学意义（P>0.05）。所有患者中位随访14（1~118）个月，98例（29.3%）复发，92例（27.5%）死亡，3年无病生存（DFS）率和总生存（OS）率分别为53.2%和58.9%。3年DFS和OS率在174例持续化疗患者中分别为34.2%和37.4%，在157例异基因造血干细胞移植患者中分别为74.5%和81.2%。多因素分析结果显示，诱导化疗时IDA剂量10 mg/m2是改善患者DFS（P=0.040）及OS（P=0.007）的独立因素。
结论: IA“3+7”方案诱导治疗初发AML患者，IDA 10 mg/m2与8 mg/m2相比，第1疗程CR率显著提高，血液学不良反应相似，并且改善DFS及OS。