The clinical impact of the molecular landscape of acute myeloid leukemia

Research into the underlying pathogenic mechanisms of acute myeloid leukemia (AML) has led to remarkable advances in our understanding of the disease. Mutations now allow us to explore the enormous diversity among cytogenetically defined subsets of AML, particularly the large subset of cytogenetical...

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Veröffentlicht in:Haematologica (Roma) Jg. 108; H. 2; S. 308 - 320
Hauptverfasser: Kayser, Sabine, Levis, Mark J.
Format: Journal Article
Sprache:Englisch
Veröffentlicht: Italy Fondazione Ferrata Storti 01.02.2023
Ferrata Storti Foundation
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ISSN:0390-6078, 1592-8721, 1592-8721
Online-Zugang:Volltext
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Zusammenfassung:Research into the underlying pathogenic mechanisms of acute myeloid leukemia (AML) has led to remarkable advances in our understanding of the disease. Mutations now allow us to explore the enormous diversity among cytogenetically defined subsets of AML, particularly the large subset of cytogenetically normal AML. Despite the progress in unraveling the tumor genome, only a small number of recurrent mutations have been incorporated into risk-stratification schemes and have been proven to be clinically relevant, targetable lesions. The current World Health Organization Classification of myeloid neoplasms and leukemia includes eight AML categories defined by recurrent genetic abnormalities as well as three categories defined by gene mutations. We here discuss the utility of molecular markers in AML in prognostication and treatment decision-making. New therapies based on targetable markers include IDH inhibitors (ivosidenib, enasidenib), venetoclax-based therapy, FLT3 inhibitors (midostaurin, gilteritinib, and quizartinib), gemtuzumab ozogamicin, magrolimab and menin inhibitors.
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Disclosures
Contributions
SK has served as a consultant for Novartis, Pfizer, Gilead and Jazz Pharmaceuticals. MJL receives research funding from Novartis and Astellas. MJL serves as a consultant for Novartis, Daiichi-Sankyo, Astellas, and Arog.
SK and MJL wrote the manuscript.
ISSN:0390-6078
1592-8721
1592-8721
DOI:10.3324/haematol.2022.280801