Acute myeloid leukemia management and research in 2025

Area of Science:

• Hematology
• Oncology
• Pharmacology

Background:

• The standard treatment for acute myeloid leukemia (AML) historically relied on cytarabine and anthracyclines.
• Significant advancements have been made with hematopoietic stem cell transplantation, high-dose cytarabine, supportive care, and targeted therapies for acute promyelocytic leukemia.

Purpose of the Study:

• To review frontline and later-line therapies for acute myeloid leukemia (AML).
• To discuss emerging research directions and novel therapeutic agents in AML treatment.

Main Methods:

• Review of recent regulatory approvals and clinical trial data for AML agents.
• Analysis of targeted therapies including BCL2 inhibitors, tyrosine kinase inhibitors, and antibody-drug conjugates.
• Discussion of novel agents like menin inhibitors and hypomethylating agents.

Main Results:

• Since 2017, 12 agents have been approved for AML treatment in the US.
• Approved agents include venetoclax, gemtuzumab ozogamicin, midostaurin, gilteritinib, quizartinib, ivosidenib, olutasidenib, enasidenib, oral azacitidine, CPX351, glasdegib, and revumenib.
• Emerging therapies such as menin inhibitors and CD123-targeting agents show promising results.

Conclusions:

• The treatment landscape for AML has dramatically evolved with numerous new drug approvals.
• Future research should focus on novel targeted therapies, antibody-drug conjugates, and combination strategies to improve outcomes in AML.