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Management of isocitrate dehydrogenase 1/2 mutated acute myeloid leukemia
Leukemia ( IF 11.4 ) Pub Date : 2024-04-10 , DOI: 10.1038/s41375-024-02246-2
Harry Fruchtman , Zachary M. Avigan , Julian A. Waksal , Nicole Brennan , John O. Mascarenhas

The emergence of next generation sequencing and widespread use of mutational profiling in acute myeloid leukemia (AML) has broadened our understanding of the heterogeneous molecular basis of the disease. Since genetic sequencing has become a standard practice, several driver mutations have been identified. Accordingly, novel targeted therapeutic agents have been developed and are now approved for the treatment of subsets of patients that carry mutations in FLT3, IDH1, and IDH2 [1, 2]. The emergence of these novel agents in AML offers patients a new modality of therapy, and shifts treatment paradigms toward individualized medicine. In this review, we outline the role of IDH mutations in malignant transformation, focus in on a novel group of targeted therapeutic agents directed toward IDH1- and IDH2-mutant AML, and explore their impact on prognosis in patients with AML.



中文翻译:

异柠檬酸脱氢酶 1/2 突变的急性髓系白血病的治疗

下一代测序的出现和突变分析在急性髓系白血病 (AML) 中的广泛使用拓宽了我们对该疾病异质性分子基础的理解。自从基因测序已成为标准做法以来,已经确定了一些驱动突变。因此,新型靶向治疗药物已被开发出来,现已被批准用于治疗携带FLT3IDH1IDH2突变的患者亚群[1, 2]。这些治疗 AML 的新型药物的出现为患者提供了一种新的治疗方式,并将治疗范式转向个体化医疗。在这篇综述中,我们概述了IDH突变在恶性转化中的作用,重点关注针对IDH1IDH2突变 AML 的一组新型靶向治疗药物,并探讨它们对 AML 患者预后的影响。

更新日期:2024-04-11
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