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Updated: Sep 22, 2025

Intracellular Phosphoflow Cytometry of Acute Myeloid Leukemia Patient-Derived Xenotransplants
Published on: June 6, 2025
Oncology stewardship in acute myeloid leukemia
Madeleine A Ochs1, Bernard L Marini1,2, Anthony J Perissinotti1
1Department of Pharmacy Services and Clinical Pharmacy, Michigan Medicine, 1540 E. Hospital Dr, Ann Arbor, MI, 48109, USA.
Abstract:
In recent years, an explosion of novel agents has shifted the treatment paradigm for patients with acute myeloid leukemia. The optimal place in therapy for many of these novel agents remains unknown due to limited guidance from national guidelines and the way these agents were studied prior to entering the market. A critical evaluation of the literature and incorporation of oncology stewardship principles can be helpful in determining an optimal place for these agents while being mindful of the overall cost that is associated with therapies. The purpose of this review is to critically evaluate the efficacy and safety data for five controversial agents and provide examples of the use of stewardship practices in determining their place in the treatment of acute myeloid leukemia.
Insights
Novel agents are changing acute myeloid leukemia (AML) treatment, but their optimal use is unclear. This review evaluates five controversial AML agents using oncology stewardship principles to guide their place in therapy.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- The treatment landscape for acute myeloid leukemia (AML) has been significantly altered by the introduction of numerous novel therapeutic agents.
- Uncertainty exists regarding the optimal integration of these new agents into clinical practice due to limited national guideline recommendations and varying study designs preceding market approval.
- The high cost associated with novel therapies necessitates careful consideration of their value and place in patient treatment regimens.
Purpose of the Study:
- To critically assess the efficacy and safety profiles of five controversial novel agents used in acute myeloid leukemia (AML) treatment.
- To explore the application of oncology stewardship principles in defining the optimal therapeutic role for these agents.
- To provide practical examples of how stewardship can inform decision-making for novel AML therapies, balancing clinical benefit with cost-effectiveness.
Main Methods:
- Comprehensive literature review focusing on efficacy and safety data for five specific novel agents in acute myeloid leukemia (AML).
- Application of established oncology stewardship principles to analyze the evaluated data.
- Case examples illustrating the integration of stewardship practices in determining the appropriate use of these agents.
Main Results:
- Efficacy and safety data for five controversial novel agents in acute myeloid leukemia (AML) were critically evaluated.
- The analysis highlighted variability in clinical benefit and toxicity across different agents and patient populations.
- Stewardship principles provided a framework for assessing the value and appropriate utilization of these therapies.
Conclusions:
- A critical evaluation of novel agents, guided by oncology stewardship, is essential for optimizing acute myeloid leukemia (AML) treatment.
- Stewardship practices can help navigate the complexities of integrating new therapies, ensuring appropriate use and cost-effectiveness.
- Further research and guideline development are needed to solidify the role of these agents in AML management.
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