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Study on Acute Myeloid Leukemia

A special issue of Cancers (ISSN 2072-6694). This special issue belongs to the section "Cancer Therapy".

Deadline for manuscript submissions: 25 May 2026 | Viewed by 11390

Special Issue Editor


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Guest Editor
Division of Pathology, The University of Texas Southwestern Medical Center, Dallas, TX 75390, USA
Interests: AML; myeloid neoplasms; biology
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Acute Myeloid Leukemia (AML) is a type of cancer that affects the blood and bone marrow, and is characterized by the rapid growth of abnormal white blood cells. The strategies employed to treat AML are developing, with chemotherapy and other therapies being utilized. AML occurs due to mutations in the hematopoietic stem cells, leading to an uncontrolled proliferation of immature blood cells. The diagnosis of AML is typically based on blood tests, bone marrow biopsy, and genetic profiling to identify mutations. In summary, this Special Issue will focus on the various treatment modalities employed to address acute myeloid leukemia. 

Dr. Olga K. Weinberg
Guest Editor

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Keywords

  • acute myeloid leukemia (AML)
  • cancer treatment
  • genetic

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Published Papers (4 papers)

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Research

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23 pages, 3202 KB  
Article
Lenalidomide Efficacy in Patients with MDS and Del-5q: Real-World Data from the Hellenic (Greek) National Myelodysplastic & Hypoplastic Syndromes Registry (EAKMYS)
by Argiris Symeonidis, Panagiotis Diamantopoulos, Athanasios Galanopoulos, Alexandra Kourakli, Eleni Sazakli, Eleftheria Hatzimichael, Maria Pagoni, Panagiotis Zikos, Theodoros P. Vassilakopoulos, Eleni Gavrilaki, Anthi Bouchla, Anna Kioumi, Katerina Palla, Ioannis Kotsianidis, Evridiki Michali, Zafiris Kartassis, Eirini Katodritou, Vasileios Lazaris, Maria Vagia, George Xanthopoulidis, Theodora Assimakopoulou, Charalampos Pontikoglou, Maria Dimou, Maria Dalekou-Tsolakou, Dimitra Liapi, Maria Kotsopoulou, Vassiliki Labropoulou, Menelaos Papoutselis, Despina Barmparousi, Efthymia Vlachaki, Georgia Kaiafa, Eleni Chandrinou, Panagiotis Karmas, Evangelos Terpos, George Vassilopoulos, Panayiotis Panayiotidis, Nora-Athina Viniou and Vassiliki Pappaadd Show full author list remove Hide full author list
Cancers 2025, 17(9), 1388; https://doi.org/10.3390/cancers17091388 - 22 Apr 2025
Cited by 4 | Viewed by 3485
Abstract
Background-Objectives: Although considered standard of care for patients with low-/intermediate-1 risk MDS and isolated del(5q), lenalidomide is not widely used in patients exhibiting additional cytogenetic abnormalities, on top of del(5)q. The aim of this study was to provide real-world evidence for the efficacy [...] Read more.
Background-Objectives: Although considered standard of care for patients with low-/intermediate-1 risk MDS and isolated del(5q), lenalidomide is not widely used in patients exhibiting additional cytogenetic abnormalities, on top of del(5)q. The aim of this study was to provide real-world evidence for the efficacy of lenalidomide in patients with del(5q), with or without additional cytogenetic abnormalities. Methods: Patients with MDS exhibiting del(5q) in the Greek National Myelodysplastic Syndromes Registry were analyzed if they had received at least one lenalidomide dose and detailed response assessment/follow-up was available. Results: Among 238 patients analyzed, 153 (64.3%) had del(5q) syndrome (Group-I), 34 (14.3%) had an isolated del(5q) abnormality but were not 5q− syndrome (Group-II), 26 (10.9%) had del(5q) plus only one additional cytogenetic abnormality (Group-III), and 25 (10.5%) had del(5q) plus >1 additional abnormality (Group-IV). Among 218 (91.6%) evaluable patients, a major response was achieved by 146 (67.0%) patients, 114/146 (78.1%) in Group-I, 18/31 (58.1%) in Group-II, 10/20 (50.0%) in Group-III, and 4/21 (19.0%) in Group-IV. Overall, hematological response was seen in 177/218 (81.2%) patients, even among those with an excess of bone marrow blasts/frank acute myeloid leukemia. Duration of response was comparable between the four patient groups. A complete cytogenetic response was achieved by 38.0% overall, more commonly in Group-I (42.3%) and -III (35.7%). Transfusion-independent patients and those with a higher MCV or lower marrow blast cells at baseline had a higher probability of achieving a major response. With multivariate analysis, factors associated with overall survival were age, performance status, transfusion dependence, and marrow blast cell percentage at treatment start, as well as time from initial diagnosis to lenalidomide start. Conclusions: Lenalidomide was highly effective in patients with the del(5)q syndrome and also in those with isolated del(5)q, other than del(5)q syndrome, or those exhibiting del(5)q plus only one additional cytogenetic abnormality, not affecting chromosome 7. Full article
(This article belongs to the Special Issue Study on Acute Myeloid Leukemia)
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14 pages, 1894 KB  
Article
Prospective, Randomized, Comparative Study of Myeloablative Fludarabine/Busulfan and Fludarabine/Busulfan/Total Body Irradiation Conditioning in Myeloid Diseases
by Hyung C. Suh, Scott D. Rowley, Sukhdeep Kaur, Brittany Lukasik, Phyllis McKiernan, Michele Boonstra, Melissa Baker, Mary DiLorenzo, Alan Skarbnik, Jason Voss, Alexandra Hampson, Bianca DeAgresta, Brighid Boylan, Themba Nyirenda, David H. Vesole and Michele L. Donato
Cancers 2025, 17(7), 1140; https://doi.org/10.3390/cancers17071140 - 28 Mar 2025
Cited by 2 | Viewed by 1361
Abstract
Background/Objectives: Allogeneic hematopoietic stem cell transplantation (alloSCT) is a curative treatment for myeloid diseases, yet relapse remains the major cause of post-transplant mortality. To reduce the risk of recurrence, we evaluated the addition of 400 cGy total body irradiation (TBI) to conditioning [...] Read more.
Background/Objectives: Allogeneic hematopoietic stem cell transplantation (alloSCT) is a curative treatment for myeloid diseases, yet relapse remains the major cause of post-transplant mortality. To reduce the risk of recurrence, we evaluated the addition of 400 cGy total body irradiation (TBI) to conditioning with fludarabine-busulfan (Flu/Bu4). Methods: In this prospective study, 46 patients with myeloid diseases were randomized to Flu/Bu4 or Flu/Bu4/TBI conditioning group. The Flu/Bu4 conditioning regimen consisted of fludarabine 40 mg/m2 on days -6 to -3 followed by busulfan 130 mg/m2 on days -6 to -3. Flu/Bu4/TBI conditioning regimen added 400 cGy TBI on day -1 to the FluBu4 regimen. Results: Among 34 acute myeloid leukemia (AML) patients, relapse was numerically lower in those who received Flu/Bu4/TBI (25%) versus Flu/Bu4 (44.4%) at three years (HR = 0.58, 95% CI 0.19 to 1.81, p = 0.35). Flu/Bu4/TBI appeared to increase the risk of non-relapse mortality (NRM) vs. Flu/Bu4 in AML patients at three years (25.0% versus 11.1%; HR = 2.11, 95% CI 0.51 to 8.83, p = 0.65). Overall survival (OS) was similar in AML patients undergoing conditioning with Flu/Bu4 (72.2%) versus Flu/Bu4/TBI (62.5%) at one year (p = 0.4). Conclusions: In conclusion, the addition of 400 cGy TBI to Flu/Bu4 reduced the risk of relapse but did not improve OS as a consequence of higher regimen-related mortality. Clinicaltrials.gov identifier: NCT01366612. Full article
(This article belongs to the Special Issue Study on Acute Myeloid Leukemia)
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Review

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54 pages, 3566 KB  
Review
Implementation of Natural Products and Derivatives in Acute Myeloid Leukemia Management: Current Treatments, Clinical Trials and Future Directions
by Faten Merhi, Daniel Dauzonne and Brigitte Bauvois
Cancers 2026, 18(2), 185; https://doi.org/10.3390/cancers18020185 - 6 Jan 2026
Viewed by 1545
Abstract
Bioactive natural products (NPs) may play a critical role in cancer progression by targeting nucleic acids and a wide array of proteins, including enzymes. Furthermore, a large number of derivatives (NPDs), including semi-synthetic products and pharmacophores from NPs, have been developed to enhance [...] Read more.
Bioactive natural products (NPs) may play a critical role in cancer progression by targeting nucleic acids and a wide array of proteins, including enzymes. Furthermore, a large number of derivatives (NPDs), including semi-synthetic products and pharmacophores from NPs, have been developed to enhance the solubility and stability of NPs. Acute myeloid leukemia (AML) is a poor-prognosis hematologic malignancy characterized by the clonal accumulation in the blood and bone marrow of myeloid progenitors with high proliferative capacity, survival and propagation abilities. A number of potential pathways and targets have been identified for development in AML, and include, but are not limited to, Fms-like tyrosine kinase 3 (FLT3) and isocitrate dehydrogenases resulting from genetic mutations, BCL2 family members, various signaling kinases and histone deacetylases, as well as tumor-associated antigens (such as CD13, CD33, P-gp). By targeting nucleic acids, FLT3 or CD33, several FDA-approved NPs and NPDs (i.e., cytarabine, anthracyclines, midostaurin, melphalan and calicheamicin linked to anti-CD33) are the major agents of upfront treatment of AML. However, the effective treatment of the disease remains challenging, in part due to the heterogeneity of the disease but also to the involvement of the bone marrow microenvironment and the immune system in favoring leukemic stem cell persistence. This review summarizes the current state of the art, and provides a summary of selected NPs/NPDs which are either entering or have been investigated in preclinical and clinical trials, alone or in combination with current chemotherapy. With multifaceted actions, these biomolecules may target all hallmarks of AML, including multidrug resistance and deregulated metabolism. Full article
(This article belongs to the Special Issue Study on Acute Myeloid Leukemia)
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17 pages, 1452 KB  
Review
Evolving Paradigms in Acute Myeloid Leukemia: Personalized Approaches to Therapy Across Age and Risk Groups
by Sumeet K. Yadav, Utsav Joshi, Guleid Hussein, Mohamed Warsame, Bolun Liu, Abhash Shrestha, Peter Krastev, Hariprasad Reddy Korsapati and Amrit Singh
Cancers 2025, 17(17), 2824; https://doi.org/10.3390/cancers17172824 - 28 Aug 2025
Cited by 2 | Viewed by 4259
Abstract
Acute myeloid leukemia (AML) is an aggressive hematologic malignancy characterized by the clonal proliferation of myeloid precursors and rapid progression. Historically consisting of intensive chemotherapy, AML management has evolved significantly due to advances in molecular diagnostics and risk stratification. This review discusses current [...] Read more.
Acute myeloid leukemia (AML) is an aggressive hematologic malignancy characterized by the clonal proliferation of myeloid precursors and rapid progression. Historically consisting of intensive chemotherapy, AML management has evolved significantly due to advances in molecular diagnostics and risk stratification. This review discusses current therapeutic paradigms in AML, emphasizing the growing role of personalized medicine across age and risk groups. For younger, fit patients, intensive regimens such as the “7 + 3” protocol remain the standard, often enhanced by targeted agents like FMS-like tyrosine kinase 3 (FLT3) and IDH inhibitors. Older or unfit individuals benefit from low-intensity treatments such as hypomethylating agents combined with venetoclax, now considered a frontline standard of care. The use of liposomal chemotherapy (CPX-351), measurable residual disease (MRD) monitoring, and maintenance therapy further refine post-remission strategies. Emerging therapies, including menin inhibitors, antibody–drug conjugates, and immunotherapies like CAR-T cells and vaccines, offer additional options, especially in relapsed/refractory settings. This comprehensive review outlines the current landscape and future directions in AML therapy, emphasizing the transition toward individualized, mutation-driven treatment strategies. Full article
(This article belongs to the Special Issue Study on Acute Myeloid Leukemia)
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