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Acute Myeloid Leukemia: Latest Advances in Diagnostics, Therapeutic Strategies and Clinical Management: 2nd Edition

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

Deadline for manuscript submissions: 20 October 2026 | Viewed by 5128

Special Issue Editor

Dr. Michael D. Diamantidis

E-Mail Website
Guest Editor
Thalassemia and Sickle Cell Disease Unit, Department of Hematology, General Hospital of Larissa, 41221 Larissa, Greece
Interests: acute myeloid leukemia; myelodysplastic syndromes; myeloid malignancies; myeloproliferative neoplasms; hemoglobinopathies
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

We are pleased to announce the 2nd Edition of this Special Issue following the great editorial success of the first Volume. You can view the papers of the first Edition through the link below:

https://www.mdpi.com/journal/jcm/special_issues/54AS713Q21

Amazing progress has been achieved in recent years regarding a better understanding of the genetic landscape of acute myeloid leukemia (AML) with the improvement of molecular diagnostics. This has led to novel classifications of the disease (ICC/ELN 2022 and WHO 2022), prognostic systems proposed by several scientific groups incorporating the genomic data of large series of AML patients with the contribution of bioinformatics, and most importantly, to the approval of novel agents for the treatment of AML. Nevertheless, there is an unmet need to solve novel challenges that have emerged.

The aim of this Special Issue is to reflect the core problems and the research regarding all aspects of AML: the genetic landscape of the disease, diagnostics, novel treatments, hematopoietic stem cell transplantation, and cellular therapies. The manuscripts submitted can be either original papers, reviews or even remarkable cases accompanied by a literature review. The basic biology of AML, novel advances regarding clonal evolution and leukemic stem cells, novel markers for diagnosis and prognosis of AML, comparison between ICC/ELN 2022 and WHO 2022 AML classifications, risk adapted therapy, novel targeted therapies, the role of measurable residual disease (MRD) in AML, current acute promyelocytic leukemia (APL) therapeutic strategies, immunotherapy of AML, CAR-T cells and AML are of interest for this Special Issue.

We thank you and we are looking forward to receiving your work.

Dr. Michael D. Diamantidis
Guest Editor

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 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-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

  • ELN
  • ICC and WHO 2022 AML classifications
  • molecular AML diagnostics
  • AML biology
  • measurable residual disease (MRD)
  • novel targeted AML therapies
  • AML immunotherapies
  • hematopoietic stem cell transplantation (HSCT)

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Related Special Issue

Published Papers (4 papers)

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Research

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11 pages, 315 KB  
Article
Real-World Utilization of Midostaurin in Combination with Intensive Chemotherapy for Patients with FLT3 Mutated Acute Myeloid Leukemia: A Multicenter Study
by Sema Seçilmiş, Sibel Kabukçu Hacıoğlu, Fehmi Hindilerden, Burhan Turgut, Düzgün Özatlı, Gülsüm Akgün Çağlıyan, Abdulkadir Baştürk, Aslı Yüksel Öztürkmen, Yavuz Katırcılar, Sinem Namdaroğlu, Başak Ünver Koluman, Cenk Sunu, Serdal Korkmaz, Ayşe Uysal, Yusuf Bilen, Mehmet Ali Erkurt, Mehmet Sinan Dal, Turgay Ulaş and Fevzi Altuntaş
J. Clin. Med. 2026, 15(2), 854; https://doi.org/10.3390/jcm15020854 - 21 Jan 2026
Viewed by 546
Abstract
Background/Objectives: Real-world data on the therapeutic use of FLT3 inhibitors in Turkey remain limited. Therefore, we retrospectively evaluated outcomes from 13 academic centers nationwide, focusing on the multikinase inhibitor midostaurin in patients with newly diagnosed FLT3-mutated acute myeloid leukemia (AML). Methods: We collected [...] Read more.
Background/Objectives: Real-world data on the therapeutic use of FLT3 inhibitors in Turkey remain limited. Therefore, we retrospectively evaluated outcomes from 13 academic centers nationwide, focusing on the multikinase inhibitor midostaurin in patients with newly diagnosed FLT3-mutated acute myeloid leukemia (AML). Methods: We collected comprehensive information regarding treatment efficacy, safety, and tolerability. Results: The overall response rate to intensive chemotherapy (3 + 7) plus midostaurin was 87.7%, with a complete remission rate of 84.2%, consistent with previously reported clinical trial results. Treatment discontinuation due to intolerance or toxicity was low (3.5%). One patient discontinued therapy because of septic shock during induction, and another due to a drug–drug interaction during consolidation. Median overall survival was 21.4 months. Allogeneic stem cell transplantation was performed in first remission in 52.6% of patients. Five patients (8.8%) were refractory to induction therapy, and relapse occurred in 21.1% (12 patients). Conclusions: These findings support the effectiveness and acceptable tolerability of midostaurin in routine clinical practice for FLT3-mutated AML. Full article
(This article belongs to the Special Issue Acute Myeloid Leukemia: Latest Advances in Diagnostics, Therapeutic Strategies and Clinical Management: 2nd Edition)
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18 pages, 2595 KB  
Article
Clinical Characteristics, Treatment Approaches, and Survival Predictors in Adult Acute Myeloid Leukemia: Interim Results from the Turkish Society of Hematology AML Registry
by Volkan Karakus, Ibrahim Ethem Pinar, Utku Iltar, Emel Merve Yenihayat, Merve Gokcen Polat, Serhat Celik, Umit Yavuz Malkan, Guldane Cengiz Seval, Ali Dogan, Aydan Akdeniz, Demircan Ozbalci, Idris Ince, Ramazan Erdem, Ozgur Mehtap, Hakki Onur Kirkizlar, Murat Kacmaz, Burak Deveci, Fatma Aykas, Gulten Korkmaz, Sureyya Yigit Kaya, Hacer Berna Afacan Ozturk, Omur Gokmen Sevindik, Ferda Can, Demet Cekdemir, Ceyda Aslan, Hale Bulbul, Zeynep Tugba Karabulut, Senem Maral, Salih Sertac Durusoy, Fatih Demirkan, Hakan Goker, Fahir Ozkalemkas, Muzaffer Keklik, Selami Kocak Toprak, Aylin Fatma Karatas, Unal Atas and Inci Alacaciogluadd Show full author list remove Hide full author list
J. Clin. Med. 2025, 14(20), 7367; https://doi.org/10.3390/jcm14207367 - 18 Oct 2025
Viewed by 1778
Abstract
Background: Acute myeloid leukemia (AML) is an aggressive and biologically diverse hematologic cancer that disproportionately affects older individuals. Despite advances in molecular profiling and therapy, long-term outcomes remain unsatisfactory. This nationwide registry was established to provide real-world insights into clinical characteristics, treatment [...] Read more.
Background: Acute myeloid leukemia (AML) is an aggressive and biologically diverse hematologic cancer that disproportionately affects older individuals. Despite advances in molecular profiling and therapy, long-term outcomes remain unsatisfactory. This nationwide registry was established to provide real-world insights into clinical characteristics, treatment strategies, and survival among adult AML patients in Turkey. Methods: The Turkish AML Registry Project (ClinicalTrials.gov Identifier: NCT05979675) combines retrospective and prospective data from 23 tertiary hematology centers. Adult patients diagnosed between January 2008 and July 2023 were included. Baseline demographics, European LeukemiaNet (ELN) 2017 risk groups, Eastern Cooperative Oncology Group (ECOG) performance status, treatment intensity, and targeted therapy use were analyzed. Response and survival outcomes were assessed using Kaplan–Meier methods. Results: The interim dataset included 891 patients (median age 58 years, 45.5% ≥60). Intensive chemotherapy, most commonly 7 + 3, was applied in 74.1%, while 25.9% received lower-intensity regimens. Targeted agents, mainly venetoclax, were incorporated more frequently into low-intensity therapies (19.1% vs. 3.4%, p < 0.001). Complete remission occurred in 70.2% after intensive and 35.9% after low-intensity therapy, improving to 51.4% with targeted agents. Median overall survival (OS) was 27.2 months, with 1-year OS rates of 54.1%, 28.9%, and 17.6% for favorable, intermediate, and adverse ELN groups (p < 0.001). ECOG 0–1 predicted superior survival (1-year OS 70.3% vs. 47.0%). Conclusions: Nationwide real-world evidence underscores the prognostic relevance of ELN risk and functional status in AML. While intensive chemotherapy remains central, combining targeted agents with low-intensity regimens improves outcomes in less fit patients and supports personalized treatment approaches. Full article
(This article belongs to the Special Issue Acute Myeloid Leukemia: Latest Advances in Diagnostics, Therapeutic Strategies and Clinical Management: 2nd Edition)
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Review

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42 pages, 1440 KB  
Review
Resistance to Targeted Therapy in AML: Current Challenges and Emerging Treatment Strategies
by Christos Stafylidis and Panagiotis T. Diamantopoulos
J. Clin. Med. 2026, 15(6), 2171; https://doi.org/10.3390/jcm15062171 - 12 Mar 2026
Viewed by 1225
Abstract
The development of targeted treatments, including inhibitors of BCL-2, FLT3, IDH1/2, and menin, has significantly expanded the therapeutic landscape of acute myeloid leukemia (AML), offering more personalized and molecularly driven treatment approaches. Despite these advances, achieving durable responses represents a major challenge, limited [...] Read more.
The development of targeted treatments, including inhibitors of BCL-2, FLT3, IDH1/2, and menin, has significantly expanded the therapeutic landscape of acute myeloid leukemia (AML), offering more personalized and molecularly driven treatment approaches. Despite these advances, achieving durable responses represents a major challenge, limited by the emergence of intrinsic and acquired resistance to targeted agents. This review summarizes the current understanding of the cellular and molecular mechanisms underlying resistance to targeted therapies in AML. Key mechanisms include acquired mutations that alter the drug target, other co-occurring genetic and epigenetic alterations, activation of bypass signaling pathways, and metabolic reprogramming. Furthermore, the role of clonal heterogeneity and the bone marrow microenvironment in the development of resistance is increasingly recognized. In addition, we discuss emerging strategies aiming at overcoming resistance, such as combination treatments and novel inhibitors designed to target resistant clones. Finally, this review highlights the critical need for mechanism-driven therapeutic design in order to achieve sustained responses and improve long-term outcomes in patients with AML. Full article
(This article belongs to the Special Issue Acute Myeloid Leukemia: Latest Advances in Diagnostics, Therapeutic Strategies and Clinical Management: 2nd Edition)
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23 pages, 1232 KB  
Review
Central Nervous System Involvement in Acute Myeloid Leukemia: From Pathophysiology to Neuroradiologic Features and the Emerging Role of Artificial Intelligence
by Rafail C. Christodoulou, Rafael Pitsillos, Vasileia Petrou, Maria Daniela Sarquis, Platon S. Papageorgiou and Elena E. Solomou
J. Clin. Med. 2026, 15(3), 1187; https://doi.org/10.3390/jcm15031187 - 3 Feb 2026
Viewed by 1172
Abstract
Background/Objectives: Central nervous system (CNS) involvement in acute myeloid leukemia (AML) is a rare but important complication linked to poor outcomes. Diagnosing it is difficult because neurological symptoms are often subtle or nonspecific, and conventional cytology and imaging have limitations. This review [...] Read more.
Background/Objectives: Central nervous system (CNS) involvement in acute myeloid leukemia (AML) is a rare but important complication linked to poor outcomes. Diagnosing it is difficult because neurological symptoms are often subtle or nonspecific, and conventional cytology and imaging have limitations. This review summarizes current evidence on the neuroradiologic features of CNS infiltration in AML and explores the growing role of artificial intelligence (AI) in enhancing detection and characterization. Methods: A thorough narrative review was conducted using PubMed, Scopus, and Embase, employing key terms related to AML, CNS involvement, MRI, CT, PET, AI, machine learning, deep learning, and radiomics. Of several thousand records, 138 relevant studies were selected and analyzed across four main areas: neuroradiologic patterns, imaging biomarkers, AI and radiomics applications, and emerging computational trends. Results: Imaging findings in AML mainly include myeloid sarcomas (isointense on T1, hyperintense on T2/FLAIR, restricted diffusion) and leptomeningeal enhancement. Secondary ischemic or hemorrhagic lesions may indicate brain leukocytosis. MRI proved more sensitive than CT, while PET/CT helped detect extramedullary disease. Recent AI and radiomics models showed high tumor classification and prognosis accuracy in similar CNS conditions, indicating significant potential for application in AML-CNS. Conclusions: Combining AI-based image analysis with multimodal neuroimaging could significantly improve diagnostic accuracy and personalized treatment for CNS involvement in AML. Progress is still challenged by the rarity of the condition and the lack of large, annotated datasets. Full article
(This article belongs to the Special Issue Acute Myeloid Leukemia: Latest Advances in Diagnostics, Therapeutic Strategies and Clinical Management: 2nd Edition)
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