急性髓系白血病（AML）的现代治疗始于1973年，首例柔红霉素和阿糖胞苷联合治疗法的成功随后拯救了大约45%的患者。准确的AML诊断依赖于形态学方法，其最初仅由细胞化学手段辅助。与急性淋巴细胞白血病（ALL）不同，至少在20世纪70年代和80年代，免疫分型在AML的诊断中几乎不起作用。可靠的细胞遗传学方法的出现为AML的预后发展带来了翻天覆地的变化。通过核型分析，可以对不同的AML实现分类与分层，以进行各种治疗。借助细胞上抗原标记物的免疫表型鉴定，独特的突变图谱可以里程碑式地进一步对 AML 患者进行分类。所有的这些进展都随着对肿瘤负荷[即微小残留病变（minimal residual disease, MRD）]的重要性的理解而成为AML患者管理的关键。MRD的疗效在过去10年迅速发展，其特异性从免疫分型的10^-3发展到聚合酶链反应（PCR）的10^-4（且仅对于部分AML患者有效），并最终在具有下一代测序（NGS）技术的灵敏度极高的细胞中发展至10^-5甚至10^-6。所有这些进步都促进了个性化医疗概念的发展，并带来了可以准确用于特定诊断亚型的靶向药物。可以精准预测与测量其响应。这些靶向药物现已成为AML管理的基础，其疗效显著提高，而毒性则显著下降。本文的重点是研究最为深入的AML靶向药物之一——FMS样酪氨酸激酶3（FLT3）抑制剂，它影响了AML的预后与治疗。作为已被批准的其他新兴靶向药物以及目前正在开发的靶向药物的原型，本文将选择性地对FLT3抑制剂展开详细讨论。

Modern therapy of acute myeloid leukemia (AML) began in 1973 with the first report of the successful combination of daunorubicin and cytarabine, which led to complete remission in approximately 45% of patients. Accurate AML diagnosis was dependent on morphology, aided initially only by cytochemistry. Unlike acute lymphoblastic leukemia (ALL), immunophenotyping offered little in the diagnosis of AML, at least during the 1970s and 1980s. The advent of reliable cytogenetics changed the entire prognostic outlook of AML. With karyotypic analysis, different groups of AML could be classified and stratified for various therapies. Unique mutational profiling was a major advance in further categorizing AML patients, aided by the immunophenotypic identification of antigenic markers on the cells. All these advances were occurring as the understanding of the importance of the tumor burden—known as minimal residual disease (MRD)—became crucial for the management of AML patients. The efficacy of MRD has rapidly progressed in the past decade, from a specificity of 10⁻³ with immunophenotyping to 10⁻⁴ with polymerase chain reaction (PCR), which is only appropriate for some patients with AML, and finally to 10⁻⁵ or even 10⁻⁶ cells with the extraordinary sensitivity of next-generation sequencing (NGS). All of these advances have promoted the concept of personalized medicine, which has led to the advent of targeted agents that can accurately be used for specific diagnostic subtypes. Responses can be predicted and measured accurately. Such targeted agents have now become a cornerstone in the management of AML, increasing efficacy and dramatically reducing toxicity. The focus of this review is on one of the most well-studied targeted agents in AML: the FMS-like tyrosine kinase 3 (FLT3) inhibitors, which have impacted the prognostication and therapeutics of AML. This review selectively discusses the FLT3 inhibitors in detail, as a model for the other burgeoning targeted agents that have already been approved, as well as those that are currently in development.