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Molecular Insights into Pediatric Cancer: Advances in Hematopoietic Stem Cell Research

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Oncology".

Deadline for manuscript submissions: 20 November 2026 | Viewed by 356

Editor


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Guest Editor
Pediatric Hematology and Oncology Department, University of Bari, Bari, Italy
Interests: pediatric oncology

Special Issue Information

Dear Colleagues,

Hematopoietic stem cells are a rare population of precursor cells with the capacity to achieve multilineage differentiation while maintaining the potential to self-renew. They are key in normal hematopoiesis and in the genesis of diseases arising in the bone marrow, since they warrant blood cell homeostasis. Understanding the biology and mechanisms that regulate normal hematopoiesis and the crosstalk between bone marrow progenitors and the microenvironment allows for a better understanding of the therapeutic potential of targeting both hematopoietic stem cells and the microenvironment in these diseases.

This Special Issue aims to showcase recent advances in our understanding of the role of the bone marrow microenvironment in normal and pathological conditions in which the derangement of hematopoiesis causes overt diseases. In pediatric age groups, myelo- and lymphoproliferative diseases mainly arise from clonal immature precursors derived from hematopoietic precursors. Hematopoietic stem cells also represent a source of treatment in different clinical conditions. Both malignant and non-malignant hematological diseases may benefit from an improved and deeper understanding of the biology of hematopoietic stem cells.

Dr. Paola Muggeo
Guest Editor

Manuscript Submission Information

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Keywords

  • bone marrow
  • microenvironment
  • precursor
  • hematopoietic stem cells
  • pediatric hematological malignancies

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Published Papers (1 paper)

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Research

21 pages, 3023 KB  
Article
Genomic Profiling, Induction Response, and Transplant Outcomes in Pediatric Acute Myeloid Leukemia: A Single-Center Retrospective Cohort Study
by Ana Maria Bicǎ, Andra Daniela Marcu, Cristina Georgiana Jercan, Iuliana Iordan, Letiția Elena Radu, Irina Avramescu, Cerasela Jardan, Dumitru Jardan, Onda Tabita Cǎlugǎru, Anda Mocanu, Andrei Colițǎ and Anca Colițǎ
Int. J. Mol. Sci. 2026, 27(13), 5832; https://doi.org/10.3390/ijms27135832 (registering DOI) - 28 Jun 2026
Abstract
Pediatric acute myeloid leukemia (AML) is biologically heterogeneous, and genomic profiling increasingly informs risk stratification and treatment. We evaluated the relationship between induction response, genomic risk, transplant allocation, and survival in pediatric AML. We retrospectively analyzed 38 pediatric patients with newly diagnosed AML, [...] Read more.
Pediatric acute myeloid leukemia (AML) is biologically heterogeneous, and genomic profiling increasingly informs risk stratification and treatment. We evaluated the relationship between induction response, genomic risk, transplant allocation, and survival in pediatric AML. We retrospectively analyzed 38 pediatric patients with newly diagnosed AML, treated between 2020 and 2025. Clinical, cytogenetic, molecular, treatment, and outcome data were collected. Genomic alterations were assessed using cytogenetics, fluorescence in situ hybridization (FISH), molecular testing, and next-generation sequencing (NGS). Survival was estimated by Kaplan–Meier analysis, and prognostic factors for event-free survival (EFS) were assessed using univariable Cox regression. This study is exploratory given the limited sample size and should be interpreted accordingly. Complete remission (CR) after the first course of induction was achieved in 25/38 patients (65.8%), partial remission (PR) in 3/38 (7.9%), and refractory disease in 10/38 (26.3%). Twenty-four patients underwent allogeneic hematopoietic stem cell transplantation; 17/24 (70.8%) were alive at last follow-up, with a 2-year overall survival rate of 72.9%. Both induction response and genomic risk stratification showed suggestive associations with outcome; descriptively, induction response showed the strongest prognostic discrimination, with achievement of CR associated with markedly improved survival. High cytogenetic risk and FLT3-ITD were significantly associated with inferior EFS. Post-induction measurable residual disease (MRD) positivity was detected in 16 of 38 patients (42.1%) and was associated with suboptimal induction response; MRD negativity did not uniformly preclude adverse outcomes, particularly in the high-risk genomic subgroup. Genomic profiling refined biological risk and post-remission treatment allocation. Integrated assessment of genomic risk, induction response, and MRD status may improve therapeutic stratification in pediatric AML. Full article
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