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Updated: May 19, 2026

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Published on: June 6, 2025
The clinically relevant pharmacogenomic changes in acute myelogenous leukemia
1University of Maryland, School of Medicine, Marlene & Stewart Greenebaum Cancer Center, Leukemia & Hematologic Malignancies, Baltimore, MD 21201, USA.
This review explores pharmacogenomics in acute myelogenous leukemia (AML) treatment, focusing on drug-metabolizing enzymes and transporters. It highlights how genetic factors influence patient response to chemotherapy and targeted therapies.
Area of Science:
- Hematology
- Pharmacogenomics
- Oncology
Background:
- Acute myelogenous leukemia (AML) is a complex cancer with diverse subtypes.
- Standard treatments like cytarabine and anthracyclines are not universally effective, especially in older adults or relapsed cases.
- Personalized medicine approaches are needed due to AML's heterogeneity.
Purpose of the Study:
- To review current knowledge on drug-metabolizing enzymes and transporters in adult AML.
- To discuss the role of pharmacogenomics in tailoring AML treatment.
- To highlight a specific gene signature for predicting response to farnesyltransferase inhibitors.
Main Methods:
- Literature review of pharmacogenomic studies in adult AML.
- Analysis of findings related to cytarabine and anthracycline metabolism and transport.
- Examination of genetic markers, including a two-gene signature (RASGRP1/APTX), for treatment response prediction.
Main Results:
- Drug-metabolizing enzymes and transporters significantly impact AML treatment efficacy.
- Pharmacogenomic variations can identify patient subsets who may benefit from specific therapies.
- A RASGRP1/APTX gene signature shows promise in predicting response to farnesyltransferase inhibitors.
Conclusions:
- Pharmacogenomic insights are crucial for optimizing AML treatment strategies.
- Personalized approaches considering genetic factors can improve outcomes for AML patients.
- Further research into pharmacogenomics will advance precision medicine in AML therapy.
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