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Review
. 2025 Jan-Feb;75(1):46-67.
doi: 10.3322/caac.21873. Epub 2024 Dec 10.

Acute myeloid leukemia management and research in 2025

Hagop M Kantarjian  1 , Courtney D DiNardo  1 , Tapan M Kadia  1 , Naval G Daver  1 , Jessica K Altman  2 , Eytan M Stein  3 , Elias Jabbour  1 , Charles A Schiffer  4 , Amy Lang  5 , Farhad Ravandi  1
Affiliations
Review

Acute myeloid leukemia management and research in 2025

Hagop M Kantarjian et al. CA Cancer J Clin. 2025 Jan-Feb.

Abstract

The first 5 decades of research in acute myeloid leukemia (AML) were dominated by the cytarabine plus anthracyclines backbone, with advances in strategies including allogeneic hematopoietic stem cell transplantation, high-dose cytarabine, supportive care measures, and targeted therapies for the subset of patients with acute promyelocytic leukemia. Since 2017, a turning point in AML research, 12 agents have received regulatory approval for AML in the United States: venetoclax (BCL2 inhibitor); gemtuzumab ozogamicin (CD33 antibody-drug conjugate); midostaurin, gilteritinib, and quizartinib (fms-like tyrosine kinase 3 inhibitors); ivosidenib, olutasidenib, and enasidenib (isocitrate dehydrogenase 1 and 2 inhibitors); oral azacitidine (a partially absorbable formulation); CPX351 (liposomal encapsulation of cytarabine:daunorubicin at a molar ratio of 5:1); glasdegib (hedgehog inhibitor); and recently revumenib (menin inhibitor; approved November 2024). Oral decitabine-cedazuridine, which is approved as a bioequivalent alternative to parenteral hypomethylating agents in myelodysplastic syndrome, can be used for the same purpose in AML. Menin inhibitors, CD123 antibody-drug conjugates, and other antibodies targeting CD123, CD33, and other surface markers are showing promising results. Herein, the authors review the frontline and later line therapies in AML and discuss important research directions.

Keywords: acute myeloid leukemia; acute promyelocytic leukemia; antibody–drug conjugate; measurable residual disease.

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Conflict of interest statement

Hagop M. Kantarjian reports research grants and honoraria from Bristol Myers Squibb Company, Daiichi‐Sankyo, Immunogen, Jazz Pharmaceuticals, and Sanofi; personal/consulting or advisory fees from AbbVie, Amgen, Astellas Pharma, Curis, Infinity BiolobiX LLC, Ipsen Biopharmaceuticals Inc., KAHR, LabCorp, Novartis, Pfizer Brazil, Shenzhen Target Rx, Stemline Therapeutics Inc., and Takeda Oncology; and support for other professional activities from Adaptive Biotechnologies, Amphista, Aptitude Health, Ascentage, BioAscend, Delta Fly, Janssen Global, and Oxford Biomedical outside the submitted work. Courtney D. DiNardo reports research support from AbbVie, Agios Pharmaceuticals Inc., Bayer, Calithera, Cleave, Bristol Myers Squibb Company, Celgene, Daiichi Sankyo Company, and ImmuneOnc; and personal/consulting fees from AbbVie, Agios Pharmaceuticals Inc., Astellas Pharma, Bristol Myers Squibb Company, Celgene, Daiichi Sankyo Company, GlaxoSmithKline, ImmuneOnc, Notable Labs, Novartis, Rigel Pharmaceuticals Inc., Servier Pharmaceuticals LLC, and Takeda Oncology; and service on a Data Safety and Monitoring Board at Genmab outside the submitted work. Tapan M. Kadia reports grants/contracts or research support from Amgen, Ascentage Pharma, Astex Pharma, AstraZeneca, Bristol Myers Squibb Company, Celgene, Genentech, Jazz Pharmaceuticals, and Pfizer Inc.; and personal/consulting fees from AbbVie, Agios Pharmaceuticals Inc., Genentech, Novartis, and Servier Pharmaceuticals LLC outside the submitted work. Naval G. Daver reports grants/contracts from Incyte Corporation, Novartis, and Sobi Inc.; personal/consulting fees from Actinium Pharmaceuticals, Agios Pharmaceuticals Inc., Amgen, Arog Pharmaceuticals, Astellas Pharma, Bristol Myers Squibb Company, Celgene, Daiichi Sankyo Company, Genentech, Gilead Sciences (aka Gilead Foundation), Gilead Sciences Inc., ImmunoGen Inc., Jazz Pharmaceuticals, Kite Pharma Inc., Novartis, Pfizer Inc., PFIZER CANADA INC., Shattuck Labs, Stemline/Menarini, Syndax, and Trillium; service on a Data Safety and Monitoring Board at Jazz Pharmaceuticals; and support for other professional activities from Astellas Pharma and Karyopharm Therapeutics outside the submitted work. Jessica K. Altman reports personal/consulting fees from AbbVie, Astellas Pharma, BlueBird Bio, Curio, Daiichi‐Sankyo Company, Gilead Sciences (aka Gilead Foundation), HMP Global, Kymera, Kura Oncology, MDEducation, PeerView, Rigel Pharmaceuticals Inc., Stemline Therapeutics Inc., and Syros; service on a Data and Safety Monitoring Board at GlycoMimetics and Kura Oncology; support for other professional activities from the National Comprehensive Cancer Network; and travel support from BioSight outside the submitted work. Eytan M. Stein reports personal/consulting fees from AbbVie, Agios Pharmaceuticals, Astellas Pharma, AstraZeneca, Celgene Corporation, Genentech, Gilead Sciences Inc., Jazz Pharmaceuticals, and Servier Pharmaceuticals LLC; and stock options in Auron Therapeutics and Scaffold Therapeutics outside the submitted work. Elias Jabbour reports personal/consulting fees from AbbVie, Adaptive Biotechnologies Corporation, Amgen, Ascentage Pharma, Autolus Ltd., Bristol Myers Squibb Company, Genentech, Novartis, PFIZER CANADA INC., Taiho Oncology Inc., Takeda Oncology, TargetRX, and TERNS Pharmaceuticals Inc. outside the submitted work. Charles A. Schiffer reports service on a Data Safety and Monitoring Board at Ascentage Pharma, Bristol Myers Squibb Company, Kartos, and Syndax; and personal/consulting fees from Bristol Myers Squibb Company and Merck outside the submitted work. Farhad Ravandi reports grants/contracts or research funding from AbbVie, Amgen, Astex, Bristol Myers Squibb Company, Macrogenics, Orsenix, Prelude, Taiho Pharmaceutical, and Xencor; personal/consulting fees from AbbVie, Agios Pharmaceuticals Inc., Amgen, Astellas Pharma, AstraZeneca, Bristol Myers Squibb Company, the Novartis Foundation, and Orsenix; and support for other professional activities from Celgene Corporation outside the submitted work. Amy Lang disclosed no conflicts of interest.

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