Abstract
目的评价高三尖杉酯碱、阿糖胞苷、柔红霉素或去甲氧柔红霉素(HAD/HAI)方案诱导治疗初治急性髓系白血病(AML)患者的长期疗效并探讨其影响因素。方法观察HAD/HAI方案治疗1个疗程后初治143例AML患者的完全缓解(CR)、总生存(OS)及无复发生存(RFS)率，分析WHO 2008标准诊断分型、遗传学预后分组及初始WBC等因素对患者OS、RFS率的影响，评估中剂量阿糖胞苷(MD-Ara-C)巩固治疗在提高AML患者长期生存中的作用。结果143例患者中112例(78.3%)1个疗程获CR。仅1例患者在诱导化疗期早期死亡。高白细胞与非高白细胞组、FLT3-ITD突变型与野生型组患者CR率差异均无统计学意义(P值分别为0.266和0.528)。遗传学预后良好、中等、不良组患者CR率分别为93.7%、71.4％和61.3%，组间差异有统计学意义(P=0.001)。中位随访24(1~104)个月，中位生存时间为30(95%CI 12~48)个月。所有患者的5年OS率为40.0%，5年RFS率为37.0%。1个疗程CR后接受巩固治疗的患者为96例，5年OS率为47.0%，5年RFS率为38.0%；其中序贯含MD-Ara-C方案(71例)和序贯标准剂量Ara-C(25例)巩固治疗组患者的5年OS率差异有统计学意义(58.0％对19.0%，P=0.004)。在序贯含MD-Ara-C方案巩固治疗的患者中，高白细胞与非高白细胞组、FLT3-ITD突变型和野生型组患者的5年OS率差异均无统计学意义(P值分别为0.419和0.482)，遗传学预后良好、中等、不良组患者5年OS率差异无统计学意义(P=0.332)。结论HAD/HAI方案诱导化疗缓解后序贯含MD-Ara-C方案巩固治疗的策略对初治AML患者，特别是高白细胞患者，可望获得满意的长期生存率。
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