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Precision Medicine in Acute Myeloid Leukemia

A special issue of Cancers (ISSN 2072-6694).

Deadline for manuscript submissions: 31 July 2026 | Viewed by 653

Special Issue Editor

Preclinical Models and Clinical Trials in Oncology and Hematology, Istituto Superiore di Sanità, Rome, Italy
Interests: acute myeloid leukemia; normal and leukemic stem cells; hematopoietic differentiation; cell differentiation therapy; targeted therapy
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Acute myeloid leukemia (AML) is a genetically heterogenous disease characterized by the impaired differentiation and increased proliferation of myeloid progenitor/precursor cells. The development of sequencing technologies has allowed tremendous progress in understanding the molecular pathogenesis of AML, thus showing great genetic and clonal heterogeneity and facilitating the identification of genetic alterations that can be targeted by specific pharmacologic agents. This has enabled the development of precision medicines, examples of which include the introduction of small molecules inhibiting FLT3, IDH1/IDH2, BCL-2 and Menin. The clinical studies carried out using these molecules in refractory/relapsed AML patients have shown moderate activity when they are used as single agents, which can be considerably potentiated by drug combination studies.

Evolving precision medicine treatment options for AML are currently in development, and it is important to emphasize the status of these treatments, focusing on the optimal selection of patients suitable for therapies, drug combinations and the stage of disease development.

For this Special Issue of Cancers, we welcome original research studies and review articles that provide an overview of the most recent advances and future challenges in the use of precision medicine in the treatment of AML patients.

Dr. Ugo Testa
Guest Editor

Manuscript Submission Information

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Keywords

  • acute myeloid leukemia
  • precision medicine
  • molecular targeting
  • DNA sequencing

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Published Papers (1 paper)

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Review

38 pages, 1794 KB  
Review
The Spectrum of Venetoclax-Based Treatments in Acute Myeloid Leukemia
by Elvira Pelosi, Germana Castelli and Ugo Testa
Cancers 2026, 18(8), 1201; https://doi.org/10.3390/cancers18081201 - 9 Apr 2026
Viewed by 442
Abstract
Background/Objectives: In recent years there has been a consistent development of clinical studies surrounding the incorporation of the B-cell lymphoma 2 (BCL-2) inhibitor venetoclax (VEN) into the treatment of acute myeloid leukemia (AML) Methods: A search of the literature showed a [...] Read more.
Background/Objectives: In recent years there has been a consistent development of clinical studies surrounding the incorporation of the B-cell lymphoma 2 (BCL-2) inhibitor venetoclax (VEN) into the treatment of acute myeloid leukemia (AML) Methods: A search of the literature showed a tremendous development of experimental and clinical studies evaluating the impact of VEN-based regimens in the treatment of AML patients. This review comprehensively analyzes the available scientific evidence—including prospective clinical trials, retrospective cohorts, and real-world studies—to summarize current knowledge on the efficacy and safety of venetoclax-based regimens in AML patients. Results: Recent studies have evaluated VEN-based regimens in newly diagnosed (ND) and refractory/relapsed (R/R) AML patients, showing the efficacy of these treatments. VEN with hypomethylating agents (HMAs) became the standard-of-care for elderly/unfit AML patients. Recent studies strongly support the effectiveness of VEN-based regimens in frontline treatment of adult AML patients eligible for intensive treatments. VEN-based therapies were also used in combination with targeted therapies, thus generating triplet therapeutic regimens that are under evaluation for the treatment of some AML subtypes. However, the response to VEN+HMAs is highly variable and in part depends on tumor genetics; some patients are resistant or relapse following VEN-based treatments and future studies will be required to develop therapeutic strategies able to circumvent resistance and to identify patients at high risk of relapse. Prospective randomized trials are required to establish the real efficacy of VEN in various clinical settings and to refine maintenance and discontinuation strategies, aiming to improve long-term outcomes and to make more safe treatments based on VEN. Full article
(This article belongs to the Special Issue Precision Medicine in Acute Myeloid Leukemia)
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