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Novel Therapeutic Strategies for Acute Myeloid Leukemia
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Novel Therapeutic Strategies for Acute Myeloid Leukemia

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Hematology".

Deadline for manuscript submissions: 15 August 2025 | Viewed by 1635

Special Issue Editors

Dr. Giovangiacinto Paterno

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Guest Editor
Hematology, Department of Biomedicine and Prevention, Tor Vergata University, 00133 Rome, Italy
Interests: acute myeloid leukemia; thrombosis and hemostasis; acute lymphoblastic leukemia
Prof. Dr. Maria Ilaria Del Principe

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Guest Editor
Hematology, Department of Biomedicine and Prevention, University Tor Vergata, 00133 Rome, Italy
Interests: acute lymphoblastic leukemia; acute myeloid leukemia; flow cytometry
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

In recent years, the development of novel molecules has significantly altered acute myeloid leukemia’s (AML) therapeutic landscape. These new drugs now allow us to provide therapeutic options to previously untreatable patients, particularly the elderly, and those resistant or refractory to conventional therapies, leading to improved outcomes. This Special Issue aims to present a detailed and up-to-date perspective on the dynamic field of AML treatment. It also focuses on how ongoing advancements in understanding AML biology are progressively leading to the development of novel drugs. At the same time, it underscores the role of supportive care, which is crucial to improving the quality of life of patients and their overall survival rates as well as managing their side effects, especially in such a fragile setting.

Dr. Giovangiacinto Paterno
Prof. Dr. Maria Ilaria Del Principe
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 short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

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-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Journal of Clinical Medicine 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 2600 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

  • acute myeloid leukemia
  • novel treatments
  • supportive care
  • quality of life
  • patients’ outcomes
  • targeted therapy

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Published Papers (2 papers)

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12 pages, 909 KiB  
Article
Use of Primary Prophylaxis with G-CSF in Acute Myeloid Leukemia Patients Undergoing Intensive Chemotherapy Does Not Affect Quality of Response
by Valeria Mezzanotte, Giovangiacinto Paterno, Ilaria Cerroni, Lucrezia De Marchi, Kristian Taka, Elisa Buzzatti, Flavia Mallegni, Elisa Meddi, Federico Moretti, Francesco Buccisano, Luca Maurillo, Raffaele Palmieri, Carmelo Gurnari, Adriano Venditti and Maria Ilaria Del Principe
J. Clin. Med. 2025, 14(4), 1254; https://doi.org/10.3390/jcm14041254 - 14 Feb 2025
Viewed by 710
Abstract
Background/Objectives: The objective of our study was to evaluate the safety and efficacy of granulocyte colony-stimulating factor (G-CSF) as primary prophylaxis in adult patients with acute myeloid leukemia (AML) undergoing intensive chemotherapy. Methods: We retrospectively analyzed 112 AML patients treated with [...] Read more.
Background/Objectives: The objective of our study was to evaluate the safety and efficacy of granulocyte colony-stimulating factor (G-CSF) as primary prophylaxis in adult patients with acute myeloid leukemia (AML) undergoing intensive chemotherapy. Methods: We retrospectively analyzed 112 AML patients treated with intensive chemotherapy at Fondazione Policlinico Tor Vergata in Rome between January 2014 and March 2024. Patients were divided into G-CSF and non-G-CSF (nG-CSF) groups. We assessed the incidence of neutropenia, its severity and duration; duration of hospitalization and its costs; incidence of febrile neutropenia (FN) and septic shock; duration of antibiotic therapy (ABT) and antifungal therapy (AFT); complete remission (CR) rates; measurable residual disease (MRD) status; relapse rates; and outcomes. Results: G-CSF administration significantly reduced the duration of neutropenia (median 14 vs. 18 days, p < 0.05) and length of hospitalization (median 28 vs. 35 days, p < 0.05), in both induction and consolidation therapy. There were no significant differences in CR rates (73% vs. 67%, p = 0.64), MRD negativity achievement (52% vs. 48%, p = 0.68), leukemia relapse rates (43% vs. 62%, p = 0.14), or overall survival (OS) (median 16.7 vs. 12.3 months, p = 0.3) between G-CSF and nG-CSF groups. Thanks to a shorter hospitalization, the use of G-CSF led to €300,000 in savings over the last 4 years. Conclusions: Our findings support the safety of G-CSF in AML patients, demonstrating no adverse impact on treatment response. G-CSF abbreviated the duration of neutropenia and hospitalization, highlighting its potential clinical and cost-effective role in AML treatment. Full article
(This article belongs to the Special Issue Novel Therapeutic Strategies for Acute Myeloid Leukemia)
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10 pages, 1126 KiB  
Article
New Combination Regimens vs. Fludarabine, Cytarabine, and Idarubicin in the Treatment of Intermediate- or Low-Risk Nucleophosmin-1-Mutated Acute Myeloid Leukemia: A Retrospective Analysis from 7 Italian Centers
by Giulia Battaglia, Davide Lazzarotto, Ilaria Tanasi, Carmela Gurrieri, Laura Forlani, Endri Mauro, Francesca Capraro, Giulia Ciotti, Eleonora De Bellis, Chiara Callegari, Luca Tosoni, Matteo Fanin, Gian Luca Morelli, Claudia Simio, Cristina Skert, Michele Gottardi, Francesco Zaja, Eleonora Toffoletti, Daniela Damiani, Renato Fanin and Mario Tiribelliadd Show full author list remove Hide full author list
J. Clin. Med. 2025, 14(3), 700; https://doi.org/10.3390/jcm14030700 - 22 Jan 2025
Viewed by 697
Abstract
Background: Nucleophosmin-1 (NPM1) mutation accounts for 30% of acute myeloid leukemia (AML) cases and defines either low- or intermediate-risk AML, depending on FLT3-ITD mutation. New combination regimens (NCRs), adding midostaurin and gemtuzumab ozogamicin (GO) to the 3 + 7 [...] Read more.
Background: Nucleophosmin-1 (NPM1) mutation accounts for 30% of acute myeloid leukemia (AML) cases and defines either low- or intermediate-risk AML, depending on FLT3-ITD mutation. New combination regimens (NCRs), adding midostaurin and gemtuzumab ozogamicin (GO) to the 3 + 7 scheme, are commonly used, though there are no data that compare NCRs with intensive induction chemotherapy. Methods: To evaluate the efficacy and safety of NCRs and FLAI in NPM1+ AML, we retrospectively analyzed 125 patients treated with FLAI (n = 53) or NCRs (n = 72) at seven Italian Centers. Results: The median age was 61 years and 51/125 (41%) were FLT3-ITD+. The complete remission (CR) rate was 77%, slightly better with NCRs (83% vs. 68%; p = 0.054). NCRs yielded a superior median overall survival (OS) (not reached (NR) vs. 27.3 months; p = 0.002), though the median event-free survival (EFS) was similar (NR vs. 20.5 months; p = 0.07). In low-risk AML, CR was higher in NCRs (94% vs. 72%, p = 0.02), as were median OS (NR vs. 41.6 months; p = 0.0002) and EFS (NR vs. 17.8 months; p = 0.0085). In intermediate-risk AML (FLT3-ITD+), there were no differences in CR (60% vs. 71%; p = 0.5), OS (p = 0.27), or EFS (p = 0.86); only allogeneic transplantation improved OS (NR vs. 13.4 months; p = 0.005), regardless of induction regimen. The safety profile was similar, except for delayed platelet recovery with FLAI (22 vs. 18 days; p = 0.0024) and higher-grade II–IV gastrointestinal toxicity with NCRs (43% vs. 18.8%; p = 0.0066). Conclusions: Our data suggest the superiority of NCRs over FLAI in low-risk patients, while all outcomes were comparable in intermediate-risk patients, a setting in which only transplants positively impacted on survival. Full article
(This article belongs to the Special Issue Novel Therapeutic Strategies for Acute Myeloid Leukemia)
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