Division of Hematology/Oncology, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Hematology Am Soc Hematol Educ Program. 2010;2010:47-55. doi: 10.1182/asheducation-2010.1.47.
Acute myeloid leukemia (AML) is a disease with marked heterogeneity in both response to therapy and survival. Cytogenetics, age, and performance status have long determined prognosis and therapy. The advent of molecular diagnostics has heralded an explosion in new prognostic factors, including gene mutations in KIT, FLT3 (Fms-like tyrosine kinase 3), NPM1 (nucleophosmin 1), and CEBPA (CCAAT enhancer-binding protein-α). Microarray technology can now identify unique gene expression signatures associated with prognosis. Similarly microRNA expression, single nucleotide polymorphism arrays, and DNA methylation signatures have recently described important new prognostic subgroups of AML, and are contributing to our understanding of AML disease biology. Combined with proteomic profiling, these technologies have helped identify new targets and signaling pathways, and may soon help to identify individual patients likely to benefit from specific therapies, including allogeneic hematopoietic cell transplantation. In summary, new clinical and molecular prognostic markers have begun to significantly improve our understanding of AML biology. We are now close to a time when we will be able to use these prognostic factors and technologies to identify new targets for therapy and to determine who may benefit from that therapy, and ultimately change how we treat individual patients with AML.
急性髓系白血病（AML）在治疗反应和生存方面存在明显的异质性。细胞遗传学、年龄和表现状态长期以来一直决定着预后和治疗。分子诊断学的出现预示着新的预后因素的爆发，包括 KIT、FLT3（Fms 样酪氨酸激酶 3）、NPM1（核仁磷酸蛋白 1）和 CEBPA（CCAAT 增强子结合蛋白-α）的基因突变。微阵列技术现在可以识别与预后相关的独特基因表达特征。同样，miRNA 表达、单核苷酸多态性阵列和 DNA 甲基化特征最近也描述了 AML 的重要新预后亚组，并有助于我们了解 AML 的疾病生物学。与蛋白质组学分析相结合，这些技术有助于确定新的靶点和信号通路，并且可能很快有助于识别可能从特定治疗中受益的个体患者，包括异基因造血细胞移植。总之，新的临床和分子预后标志物开始显著改善我们对 AML 生物学的理解。我们现在即将能够使用这些预后因素和技术来确定新的治疗靶点，并确定谁可能从该治疗中受益，并最终改变我们治疗 AML 个体患者的方式。
