Future Perspectives for Treatment and Diagnosis of Acute Myeloid Leukemia (AML)

A special issue of Current Oncology (ISSN 1718-7729). This special issue belongs to the section "Hematology".

Deadline for manuscript submissions: 30 November 2025 | Viewed by 407

Special Issue Editors


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Guest Editor
1. Division of Hematology, Department of Medicine, University of Ottawa, Ottawa, ON K1N 6N5, Canada
2. The Ottawa Hospital Research Institute, Ottawa, ON K1N 6N5, Canada
Interests: acute leukemia; myelodysplastic neoplasms; bone marrow failure syndromes

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Guest Editor
Department of Medicine, McMaster University, Hamilton, ON L8N 3Z5, Canada
Interests: asciminib; acute myeloid leukemia; myelodysplastic syndromes; dasatinib

Special Issue Information

Dear Colleagues,

Until recently, treatment for acute myeloid leukemia was only offered to the most fit patients (usually <70 years of age), even though most patients are over the age of 70 years. However, with the improved understanding of its pathophysiology, over the past 10–20 years, new targets and biomarkers have been identified that are prognostic and/or predictive of therapy. These have helped to improve the outcome of disease in both fit and less-fit patients, broadening access to therapy and improving outcomes for specific subtypes. The further expansion of this understanding continues to add possible new targets and/or monitoring and intervention strategies to further improve the outcome of the disease.

In this Special Issue, original research articles and reviews are welcome. I look forward to receiving your contributions.

Dr. Mitchell Sabloff
Prof. Dr. Brian Leber
Guest Editors

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Keywords

  • pathophysiology
  • biomarker
  • diagnosis/prognosis/predictive/monitoring (indication/limitations)
  • treatment advances
  • intensive
  • less intensive
  • refractory leukemia
  • cellular therapies

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Published Papers (2 papers)

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12 pages, 500 KiB  
Article
Clinical Effects of RUNX1 Mutations on the Outcomes of Patients with Acute Myeloid Leukemia Treated with Allogeneic Hematopoietic Stem-Cell Transplantation
by Wei-Jie Ran, Lan-Ping Xu, Xiao-Hui Zhang, Ying-Jun Chang, Xiao-Dong Mo, Yu-Qian Sun, Xiao-Jun Huang and Yu Wang
Curr. Oncol. 2025, 32(6), 294; https://doi.org/10.3390/curroncol32060294 - 22 May 2025
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Abstract
It is reported that AML with RUNX1 mutations is associated with poorer response to conventional chemotherapy, lower rates of complete remission (CR), leukemia-free survival (LFS), and overall survival (OS). We aimed to evaluate the prognostic impact of RUNX1 mutations following allogeneic hematopoietic stem [...] Read more.
It is reported that AML with RUNX1 mutations is associated with poorer response to conventional chemotherapy, lower rates of complete remission (CR), leukemia-free survival (LFS), and overall survival (OS). We aimed to evaluate the prognostic impact of RUNX1 mutations following allogeneic hematopoietic stem cell transplantation (allo-HSCT) by comparing clinical outcomes in AML patients with and without RUNX1 mutations. We retrospectively analyzed 91 AML patients (33 RUNX1+ and 58 RUNX1−) who received their first HSCT at Peking University People’s Hospital. The median age of the cohort was 38 years (range: 6–64), with 73 patients (80%) receiving Haploidentical HSCT and 18 patients (20%) receiving sibling-matched allo-HSCT. In univariate analyses, no significant differences in survival outcomes were observed. For the RUNX1-mutation group and RUNX1-wild-type group, the 2-year cumulative incidence of relapse (CIR) was (12.6% vs. 7.6%, p = 0.472), the 2-year non-relapse mortality (NRM) rate was (9.6% vs. 7.2%, p = 0.747), the 2-year LFS was (77.8% vs. 85.2%, p = 0.426), and the 2-year OS rate was (85.9% vs. 92.7%, p = 0.397). We did not observe any negative impact of RUNX1 mutations on clinical outcomes, suggesting that allo-HSCT (especially Haplo-HSCT) may mitigate the adverse prognostic influence of RUNX1 mutations in AML. Full article
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24 pages, 1075 KiB  
Systematic Review
CAR-T Cell Therapy for Acute Myeloid Leukemia: Where Do We Stand Now?
by Pilar Lloret-Madrid, Pedro Chorão, Manuel Guerreiro and Pau Montesinos
Curr. Oncol. 2025, 32(6), 322; https://doi.org/10.3390/curroncol32060322 - 30 May 2025
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Abstract
Background: Patients with refractory and relapsed acute myeloid leukemia (R/R AML) face a dismal prognosis. CAR-T therapy has emerged as a potential treatment option. This study assesses the available clinical evidence on CAR-T in R/R AML, focusing on safety and efficacy outcomes. [...] Read more.
Background: Patients with refractory and relapsed acute myeloid leukemia (R/R AML) face a dismal prognosis. CAR-T therapy has emerged as a potential treatment option. This study assesses the available clinical evidence on CAR-T in R/R AML, focusing on safety and efficacy outcomes. Methods: We included studies on CAR-T therapy for R/R AML published from June 2014 to January 2025. Data on patient and disease characteristics, CAR-T constructs, response rates, post-CAR-T allogeneic HSCT (allo-HSCT), and safety outcomes were analyzed. Results: Twenty-five CAR-T clinical trials involving 296 patients were identified. The most frequently targeted antigens were CD33, CD123, and CLL-1, while CD7, CD19, NKG2D, and CD38 were also explored. Responses were heterogeneous and often short-lived when not consolidated with allo-HSCT. Cytokine release syndrome and neurotoxicity were generally low grade and manageable. Prolonged and severe myelosuppression was a frequent limiting toxicity, often requiring allo-HSCT to restore hematopoiesis. Disease progression was the leading cause of death, followed by infections. Conclusions: CAR-T cell therapy may represent a feasible therapeutic strategy, particularly as bridging to allo-HSCT to mitigate myelotoxicity and improve long-term outcomes. Nevertheless, it remains in the early stages of development and faces significant efficacy and safety challenges that must be addressed in future trials to enable the expansion of this promising therapeutic approach for a population with high unmet medical needs. Full article
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