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Updated: Nov 5, 2025

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Towards precision medicine for AML.

Hartmut Döhner1, Andrew H Wei2, Bob Löwenberg3,4

  • 1Department of Internal Medicine III, University of Ulm, Ulm, Germany. hartmut.doehner@uniklinik-ulm.de.

Nature Reviews. Clinical Oncology
|May 19, 2021
PubMed
Summary
This summary is machine-generated.

Precision medicine advances in acute myeloid leukaemia (AML) utilize targeted inhibitors and combinations for better outcomes. Challenges include drug resistance and clinical trial design for novel immunotherapies.

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Area of Science:

  • Hematology
  • Oncology
  • Molecular Biology

Background:

  • Advances in sequencing technologies have elucidated the molecular pathogenesis of acute myeloid leukaemia (AML).
  • Significant genetic and clonal heterogeneity exists within AML, necessitating tailored treatment strategies.
  • Precision medicine has emerged as a promising approach for AML management.

Purpose of the Study:

  • To review the current landscape of precision medicine in AML.
  • To discuss the efficacy of targeted inhibitors and combination therapies.
  • To highlight challenges and future directions in AML treatment, including immunotherapy.

Main Methods:

  • Review of current literature on precision medicine in AML.
  • Analysis of targeted therapies (FLT3, IDH1/IDH2, BCL-2 inhibitors) and their combinations.
  • Discussion of emerging immunotherapies and their clinical development.

Main Results:

  • Targeted inhibitors (FLT3, IDH1/IDH2, BCL-2) show moderate activity as single agents.
  • Combination therapies involving targeted inhibitors, epigenetic modulators, or cytotoxic agents enhance clinical activity.
  • Immunotherapies for AML face challenges due to antigen expression on normal hematopoietic cells but are advancing.

Conclusions:

  • Precision medicine, particularly targeted therapies and combinations, holds significant potential to improve AML patient outcomes.
  • Overcoming resistance mechanisms and optimizing clinical trial designs are crucial for future AML treatment.
  • Continued research into novel agents and combination strategies is essential for advancing AML care.