cancers-logo

Journal Browser

Journal Browser

Clinical Treatment and Translational Advances in Myelodysplastic Syndromes and Acute Myeloid Leukemia

A Special Issue of Cancers (ISSN 2072-6694).

Deadline for manuscript submissions: 30 November 2026 | Viewed by 2260

Editors


E-Mail Website
Guest Editor
1. Department of Biomedicine and Prevention, University of Rome Tor Vergata, 00133 Rome, Italy
2. UniCamillus-Saint Camillus International University of Health Sciences, Rome, Italy
Interests: de novo, secondary, and therapy-related myeloid neoplasms: individual susceptibility, risk factors, diagnostic/prognostic framework, and identification of new molecular targets and markers of treatment response

E-Mail Website
Guest Editor
Hematology, Department of Biomedicine and Prevention, University of Rome Tor Vergata, 00133 Rome, Italy
Interests: acute myeloid leukemia; myelodysplastic syndromes; myeloproliferative neoplasms; acute promyelocytic leukemia
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Myeloid neoplasms (MN), including myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), are heterogeneous malignancies ranging from indolent chronic diseases to aggressive high-risk phenotypes. In recent years, major advances in the biological understanding of MN have significantly deepened our knowledge of the molecular drivers of disease evolution, treatment resistance, and relapse.

On the clinical side, important therapeutic breakthroughs have reshaped—and will continue to modify in the near future—the management of several MN subgroups. In lower-risk MDS, novel agents such as luspatercept, a TGF-β ligand trap, and imetelstat, a telomerase inhibitor, have demonstrated remarkable efficacy in transfusion-dependent patients. Similarly, the addition of venetoclax to intensive chemotherapy in refractory or very high-risk AML has yielded highly promising results. Targeted therapies have also expanded our armamentarium: menin inhibitors for AML with NPM1 mutations or KMT2A rearrangements, and IDH1/IDH2 inhibitors for IDH-mutated AML, are among the most notable innovations. Conversely, therapeutic progress in high-risk MDS has lagged behind. Since the introduction of hypomethylating agents (HMAs), no major breakthroughs have emerged, and recent trials combining HMAs with venetoclax, tamibarotene, sabatolimab, pevonedistat, or eprenetapopt have not demonstrated superior complete remission rates or overall survival compared to monotherapy.

This Special Issue aims to highlight recent advances across all aspects of MDS and AML management, encompassing therapeutic innovations, translational research, biomarker discovery, and novel prognostic models. We warmly welcome inquiries and encourage the submission of original research articles and comprehensive reviews aligned with the scope of this Issue.

Prof. Dr. Emiliano Fabiani
Dr. Luca Guarnera
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-anonymized peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Cancers is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • myeloid neoplasms
  • myelodysplastic neoplasms
  • acute myeloid leukemia
  • leukemogenesis
  • target therapy

Benefits of Publishing in a Special Issue

  • Ease of navigation: Grouping papers by topic helps scholars navigate broad scope journals more efficiently.
  • Greater discoverability: Special Issues support the reach and impact of scientific research. Articles in Special Issues are more discoverable and cited more frequently.
  • Expansion of research network: Special Issues facilitate connections among authors, fostering scientific collaborations.
  • External promotion: Articles in Special Issues are often promoted through the journal's social media, increasing their visibility.
  • Reprint: MDPI Books provides the opportunity to republish successful Special Issues in book format, both online and in print.

Further information on MDPI's Special Issue policies can be found here.

Published Papers (2 papers)

Order results
Result details
Select all
Export citation of selected articles as:

Research

Jump to: Review

17 pages, 1432 KB  
Article
Azacitidine Is Well-Tolerated and Is Associated with High Response Rate in Elderly Patients with Higher-Risk Myelodysplastic Syndromes: A Single Center Observational Study
by Nupur Krishnan, David Yanni, Leah Kogan, Lauren Gerard, Jesse McLean and Rouslan Kotchetkov
Cancers 2026, 18(13), 2131; https://doi.org/10.3390/cancers18132131 - 30 Jun 2026
Viewed by 484
Abstract
Background/Objectives: Azacitidine (AZA) is the standard of care for patients with higher-risk Myelodysplastic Syndromes (MDS). There is limited real-life data, however, characterizing its efficacy and safety profile in elderly patients. Methods: We conducted a single-center retrospective cohort chart review to compare front-line AZA [...] Read more.
Background/Objectives: Azacitidine (AZA) is the standard of care for patients with higher-risk Myelodysplastic Syndromes (MDS). There is limited real-life data, however, characterizing its efficacy and safety profile in elderly patients. Methods: We conducted a single-center retrospective cohort chart review to compare front-line AZA therapy in patients ≥75 years (elderly) vs. <75 years (younger) with higher-risk MDS treated at our cancer center. The primary endpoint was overall survival and main secondary endpoints included response, as per the 2006 International Working Group consensus criteria, leukemia-free survival, transfusion independence, and safety outcomes. Results: In total, 55 patients were elderly (median age: 79.9 years), including 27 patients >80 years, and 41 were younger (median age: 69.4 years). Baseline demographic variables were similar between both groups. The majority of elderly patients (98%) received the full dose of AZA (75 mg/m2), compared with 90% of younger patients. The median number of AZA cycles was 8 (range: 2–69) in elderly and 7.75 (range: 1–96) in younger patients. Treatment delays occurred in 36.4% of elderly and 29.3% of younger patients, most commonly due to infection complications in both groups (p = 0.076). Disease control rates (complete remission + partial remission + stable disease) were 92.9% in the younger subgroup and 96.4% in the elderly subgroup (p = 0.154). Relapse occurred in 48.8% of younger patients and 40.0% of elderly patients. Median overall survival (OS) was 17.3 months for the younger subgroup, 15.7 months for the elderly subgroup (p = 0.771), and 11.9 months among patients >80 years (p = 0.381). Mortality rates and causes of death were similar between both subgroups. Most common causes of death included disease progression, sepsis, febrile neutropenia, and pneumonia. Conclusions: AZA monotherapy resulted in a high response rate and was well-tolerated in elderly patients with higher-risk MDS. These findings remain consistent in the real-world setting despite potential confounding factors that may contribute to inferior outcomes. Full article
Show Figures

Figure 1

Review

Jump to: Research

21 pages, 750 KB  
Review
Epigenetic Regulation in Acute Myeloid Leukemia: Molecular Mechanisms and Clinical Implications
by Jingru Xu and Georges Lacaud
Cancers 2026, 18(14), 2203; https://doi.org/10.3390/cancers18142203 - 8 Jul 2026
Viewed by 1308
Abstract
Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy characterized by a block of differentiation and uncontrolled expansion of myeloid progenitor cells. Standard treatment includes intensive induction chemotherapy, typically with cytarabine and anthracycline, followed by consolidation chemotherapy or allogeneic hematopoietic stem cell transplantation. [...] Read more.
Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy characterized by a block of differentiation and uncontrolled expansion of myeloid progenitor cells. Standard treatment includes intensive induction chemotherapy, typically with cytarabine and anthracycline, followed by consolidation chemotherapy or allogeneic hematopoietic stem cell transplantation. However, these approaches are often associated with relapse and treatment-related toxicity. Accumulating evidence highlights a critical role for epigenetic dysregulation in driving disease initiation, progression, and therapeutic resistance. In this review, we examine an integrated framework of epigenetic regulation in AML, encompassing DNA methylation, histone post-translational modifications, chromatin remodeling, and RNA-mediated epigenetics. We discuss how alterations in key epigenetic regulators, such as DNMT3A, TET2, IDH1/2, EZH2, and histone-modifying enzymes, reshape the transcriptional and epigenetic landscape of leukemic cells. Particular emphasis is placed on epigenetically defined AML subtypes, including NPM1-mutated, DNMT3A-mutated, and KMT2A-rearranged AML, which illustrate distinct mechanisms of transcriptional and epigenetic dysregulation and confer unique therapeutic vulnerabilities. We further summarize current and emerging therapeutic strategies, ranging from conventional chemotherapy to molecularly targeted agents, epigenetic drugs, and immunotherapeutic approaches. Despite these advances, durable responses remain limited, highlighting the need to better understand epigenetic mechanisms to overcome resistance and improve patient outcomes. Full article
Show Figures

Figure 1

Back to TopTop