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Children
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Pediatric Acute Myeloid Leukemia: Current Management and Future Directions
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Pediatric Acute Myeloid Leukemia: Current Management and Future Directions

A special issue of Children (ISSN 2227-9067). This special issue belongs to the section "Pediatric Hematology & Oncology".

Deadline for manuscript submissions: closed (31 December 2019) | Viewed by 33299

Special Issue Editors

Dr. Michele S. Redell

E-Mail Website
Guest Editor
Baylor College of Medicine
Interests: pediatric AML; chemotherapy resistance; microenvironment; signal transduction; novel agents; mouse models
Dr. Alexandra M. Stevens

E-Mail Website
Guest Editor
Baylor College of Medicine
Interests: pediatric AML; risk stratification; microenviroment; chemotherapy resistance; targeted agents; tumor metabolism; mouse models

Special Issue Information

Dear Colleagues,

While tremendous advances have been made in improving outcomes for children with cancer in general, progress for children with acute myeloid leukemia (AML) has been relatively slow. Chemotherapy intensity has been pushed to the limit of tolerability, and still, nearly 40% of children will suffer relapse. As we expand our understanding of the biology of the disease, we expect that new approaches to risk stratification, therapy, and supportive care will emerge. This Special Issue of Children will bring together the most current basic, translational, and clinical research focused on this challenging disease. Both reviews and original research will be considered for publication. Basic and translational research topics could include genomic/transcriptomic/proteomic studies, small molecule therapies, immunotherapies, and biology studies with clinical samples. Clinical research topics could include new approaches to upfront treatment, minimal residual disease detection, stem cell transplantation, salvage therapy, and management of AML in resource-poor settings.

Dr. Michele S. Redell
Dr. Alexandra M. Stevens
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. Children is an international peer-reviewed open access monthly 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 2400 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
  • structural rearrangement
  • microenvironment
  • risk stratification
  • epigenetic mechanisms of leukemogenesis
  • chemotherapy resistance
  • kinase inhibitors
  • immunotherapy
  • stem cell transplant
  • opportunistic infections
  • supportive care

Published Papers (6 papers)

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Editorial

Jump to: Research, Review, Other

2 pages, 139 KiB  
Editorial
Introduction to the Special Issue on Pediatric Acute Myeloid Leukemia: Current Management and Future Directions
by Alexandra M. Stevens and Michele S. Redell
Children 2021, 8(8), 698; https://doi.org/10.3390/children8080698 - 12 Aug 2021
Viewed by 1276
Abstract
This Special Issue brings together an original research report, a fascinating case report, and three timely reviews on a variety of topics related to pediatric leukemia [...] Full article
(This article belongs to the Special Issue Pediatric Acute Myeloid Leukemia: Current Management and Future Directions)

Research

Jump to: Editorial, Review, Other

15 pages, 798 KiB  
Article
The Developmental Pathways of Preschool Children with Acute Lymphoblastic Leukemia: Communicative and Social Sequelae One Year after Treatment
by Marta Tremolada, Livia Taverna, Sabrina Bonichini, Marta Pillon and Alessandra Biffi
Children 2019, 6(8), 92; https://doi.org/10.3390/children6080092 - 13 Aug 2019
Cited by 5 | Viewed by 5072
Abstract
Early childhood is considered to be a period of rapid development, with the acquisition of abilities predicting future positive school competences. Motor, cognitive, and social difficulties related to cancer therapies heavily impact the development of children with cancer. This study focused on two [...] Read more.
Early childhood is considered to be a period of rapid development, with the acquisition of abilities predicting future positive school competences. Motor, cognitive, and social difficulties related to cancer therapies heavily impact the development of children with cancer. This study focused on two main aims: To assess the developmental pathways of preschool children with acute lymphoblastic leukemia one year post-treatment and to compare these abilities both with those of a control group of healthy peers and with Italian norms. Forty-four children and their families, recruited through the Hematology-Oncologic Clinic of the Department of Child and Woman Health (University of Padua), agreed to participate in this study. The children’s mean age was 4.52 years (SD = 0.94, range = 2.5–6 years), equally distributed by gender, all diagnosed with acute lymphoblastic leukemia. Matched healthy peers were recruited through pediatricians’ ambulatories. Each family was interviewed adopting the Vineland adaptive behavior scales. Paired sample Wilcoxon tests revealed that children were reported to have significantly more developmental difficulties than their healthy peers. When compared with Italian norms, they scored particularly low in verbal competence, social, and coping skills. No significant association was found between treatment variables and developmental abilities. These findings suggest that the creation of specialized interventions, both for parents and children, may fill the possible delays in children’s development probably due to stress, lack of adequate stimulation, or difficult adaptation. Full article
(This article belongs to the Special Issue Pediatric Acute Myeloid Leukemia: Current Management and Future Directions)
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Review

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15 pages, 648 KiB  
Review
Harnessing T Cells to Target Pediatric Acute Myeloid Leukemia: CARs, BiTEs, and Beyond
by Rebecca Epperly, Stephen Gottschalk and Mireya Paulina Velasquez
Children 2020, 7(2), 14; https://doi.org/10.3390/children7020014 - 17 Feb 2020
Cited by 15 | Viewed by 6504
Abstract
Outcomes for pediatric patients with acute myeloid leukemia (AML) remain poor, highlighting the need for improved targeted therapies. Building on the success of CD19-directed immune therapy for acute lymphocytic leukemia (ALL), efforts are ongoing to develop similar strategies for AML. Identifying target antigens [...] Read more.
Outcomes for pediatric patients with acute myeloid leukemia (AML) remain poor, highlighting the need for improved targeted therapies. Building on the success of CD19-directed immune therapy for acute lymphocytic leukemia (ALL), efforts are ongoing to develop similar strategies for AML. Identifying target antigens for AML is challenging because of the high expression overlap in hematopoietic cells and normal tissues. Despite this, CD123 and CD33 antigen targeted therapies, among others, have emerged as promising candidates. In this review we focus on AML-specific T cell engaging bispecific antibodies and chimeric antigen receptor (CAR) T cells. We review antigens being explored for T cell-based immunotherapy in AML, describe the landscape of clinical trials upcoming for bispecific antibodies and CAR T cells, and highlight strategies to overcome additional challenges facing translation of T cell-based immunotherapy for AML. Full article
(This article belongs to the Special Issue Pediatric Acute Myeloid Leukemia: Current Management and Future Directions)
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Figure 1

15 pages, 3983 KiB  
Review
New and Emerging Targeted Therapies for Pediatric Acute Myeloid Leukemia (AML)
by Jing Chen and Chana L. Glasser
Children 2020, 7(2), 12; https://doi.org/10.3390/children7020012 - 10 Feb 2020
Cited by 23 | Viewed by 8931
Abstract
The relapse rate for children with acute myeloid leukemia (AML) remains high despite advancements in risk classification, multi-agent chemotherapy intensification, stem cell transplantation, and supportive care guidelines. Prognosis for this subgroup of children with relapsed/refractory AML remains poor. It is well known that [...] Read more.
The relapse rate for children with acute myeloid leukemia (AML) remains high despite advancements in risk classification, multi-agent chemotherapy intensification, stem cell transplantation, and supportive care guidelines. Prognosis for this subgroup of children with relapsed/refractory AML remains poor. It is well known that the ceiling of chemotherapy intensification has been reached, limited by acute and chronic toxicity, necessitating alternative treatment approaches. In the last several years, our improved understanding of disease biology and critical molecular pathways in AML has yielded a variety of new drugs to target these specific pathways. This review provides a summary of antibody drug conjugates (ADCs), small molecule inhibitors, and tyrosine kinase inhibitors with an emphasis on those that are currently under clinical evaluation or soon to open in early phase trials for children with relapsed/refractory AML. Full article
(This article belongs to the Special Issue Pediatric Acute Myeloid Leukemia: Current Management and Future Directions)
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Figure 1

11 pages, 243 KiB  
Review
Advances in Pediatric Acute Promyelocytic Leukemia
by Shannon E. Conneely and Alexandra M. Stevens
Children 2020, 7(2), 11; https://doi.org/10.3390/children7020011 - 2 Feb 2020
Cited by 20 | Viewed by 6312
Abstract
Acute promyelocytic leukemia (APL) is a rare disease accounting for only 5%–10% of pediatric acute myeloid leukemia (AML) and fewer than 1000 cases occur annually in the United States across all age groups. Characterized by t (15; 17), with a resultant PML-RARA gene [...] Read more.
Acute promyelocytic leukemia (APL) is a rare disease accounting for only 5%–10% of pediatric acute myeloid leukemia (AML) and fewer than 1000 cases occur annually in the United States across all age groups. Characterized by t (15; 17), with a resultant PML-RARA gene fusion driving leukemia development, advances in therapy have improved outcomes for APL significantly in the past several decades, now making APL the most curable form of AML in both children and adults. Cure rates in APL are now comparable to pediatric B-lymphoid leukemias. The success of APL treatment is due, in part, to the breadth of understanding of the driver PML-RARA mutation as well as collaborative efforts to quickly introduce and maximize the benefit of new therapies. Here, we review the presentation, clinical features, pathogenesis, and treatment advances in pediatric APL. Full article
(This article belongs to the Special Issue Pediatric Acute Myeloid Leukemia: Current Management and Future Directions)

Other

5 pages, 188 KiB  
Case Report
Transient Abnormal Myelopoeisis and Mosaic down Syndrome in a Phenotypically Normal Newborn
by Zachary Prudowsky, HyoJeong Han and Alexandra Stevens
Children 2020, 7(6), 52; https://doi.org/10.3390/children7060052 - 28 May 2020
Cited by 7 | Viewed by 4558
Abstract
Transient abnormal myelopoiesis (TAM) is a common and potentially fatal neonatal complication of newborn babies with Down syndrome (DS). Children born with mosaic DS are also at risk of developing TAM. However, due to their variable phenotypes, early identification of patients with mosaic [...] Read more.
Transient abnormal myelopoiesis (TAM) is a common and potentially fatal neonatal complication of newborn babies with Down syndrome (DS). Children born with mosaic DS are also at risk of developing TAM. However, due to their variable phenotypes, early identification of patients with mosaic DS may be difficult; thus, early diagnosis of TAM is just as challenging. In this report, we describe a case of a phenotypically normal newborn who presented with concerns for neonatal leukemia. The diagnosis of mosaic DS and TAM was confirmed with abnormal GATA1 mutation testing, highlighting the importance of early GATA1 mutation testing in newborn leukemia with high suspicion for TAM. Full article
(This article belongs to the Special Issue Pediatric Acute Myeloid Leukemia: Current Management and Future Directions)
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