DH1R132, DH2R140 and 1DH2R172 in AML: different genetic landscapes correlate with outcome and may influence targeted treatment strategies
DH1R132, DH2R140 and 1DH2R172 in AML: different genetic landscapes correlate with outcome and may influence targeted treatment strategies
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DOI：
10.1038/s41375-018-0026-z
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发表时间：
2018-05-01
期刊：
影响因子：
11.4
通讯作者：
Haferlach, Torsten
中科院分区：
文献类型：
--
作者：
Meggendorfer, Manja;Cappelli, Luca Vincenzo;Haferlach, Torsten
急性髓系白血病 (AML) 的特征是未分化骨髓前体细胞克隆扩张导致造血受损和骨髓衰竭。 AML 的分类主要由细胞形态学、细胞遗传学和单基因突变（如 NPM1、CEBPA 或 RUNX1）驱动。精准医学旨在专门针对不同的基因突变。在 AML 中，有五种靶向疗法已被使用或正在临床研究中。全反式维甲酸 (ATRA) 的成熟疗法针对 PML-RARA 突变的 AML，米斯托林针对 FLT3 突变的 AML，而仍在研究中
Acute myeloid leukemia (AML) is characterized by impaired hematopoiesis and bone marrow failure caused by clonal expansion of undifferentiated myeloid precursors. The classification of AML is mainly driven by cytomorphology, cytogenetics and single gene mutations like NPM1, CEBPA or RUNX1. Precision medicine aims at specifically targeting different gene mutations. In AML, five targeted therapies are used or under clinical investigation. The well-established therapy with all-trans retinoic acid (ATRA) targets PML-RARA-mutated AML, midostaurin targets FLT3-mutated AML, while the still investigated