Molecular genetic pathways as therapeutic targets in acute myeloid leukemia.
Molecular genetic pathways as therapeutic targets in acute myeloid leukemia.
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DOI：
10.1182/asheducation-2008.1.400
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发表时间：
2008-01-01
期刊：
影响因子：
--
通讯作者：
Haferlach, Torsten
中科院分区：
文献类型：
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作者：
Haferlach, Torsten
急性髓性白血病（AML）的异质性是由细胞遗传畸变和分子突变的复杂网络引起的。这些遗传标记是在不同亚组中对病例进行分类的基础，与AML的预后预测和治疗决策高度相关。不同遗传定义亚群的临床差异可能部分与不同突变类别的相互作用有关，并且基于复发性分子突变的正常核型AML的细分与治疗决策的相关性越来越大。与这些关于AML遗传网络复杂性的重要见解同时，临床前和临床研究中正在研究各种不同的新化合物。这些方法旨在开发基于干扰分子遗传或表观遗传机制的靶向治疗概念。本文综述了目前或将来可作为靶向治疗方法基础的最相关的遗传标记，并总结了靶向治疗的最新研究结果。
The heterogeneity of acute myeloid leukemia (AML) results from a complex network of cytogenetic aberrations and molecular mutations. These genetic markers are the basis for the categorization of cases within distinct subgroups and are highly relevant for the prediction of prognosis and for therapeutic decisions in AML. Clinical variances within distinct genetically defined subgroups could in part be linked to the interaction of diverse mutation classes, and the subdivision of normal karyotype AML on the basis of recurrent molecular mutations gains increasing relevance for therapeutic decisions. In parallel to these important insights in the complexity of the genetic networks in AML, a variety of diverse new compounds is being investigated in preclinical and clinical studies. These approaches aim to develop targeted treatment concepts that are based on interference with molecular genetic or epigenetic mechanisms. This review provides an overview on the most relevant genetic markers, which serve as basis for targeted therapy approaches now or might represent options for such approaches in the future, and summarizes recent results of targeted therapy studies.