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Acute Leukemias in Children: Existing Challenges and Emerging Therapies
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Acute Leukemias in Children: Existing Challenges and Emerging Therapies

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

Deadline for manuscript submissions: closed (15 July 2022) | Viewed by 10857

Special Issue Editor

Dr. Miguel Angel Diaz

E-Mail Website
Guest Editor
Department of Pediatrics, Hospital Infantil Universitario “Niño Jesús”, Madrid, Spain
Interests: hematopoietic stem cell transplantation; cellular therapy; pediatric hematological malignancies

Special Issue Information

Dear Colleagues,

Over the past decade, we have witnessed revolutionary changes in the treatment of pediatric acute leukemias. Nowadays, the probability of cure for pediatric acute lymphoblastic leukemia (ALL) patients is around 90% with risk-adapted modern chemotherapy. However, the outcomes of patients with pediatric acute myeloid leukemia or relapsed ALL are not as successful. The role of allogeneic HSC transplantation for those patients is well stablished, but relapse disease is by far the main cause of transplant failure. As pediatricians, we are now facing emerging knowledge and therapies such as new insights into the biology of the disease, targeted-adapted immunotherapies, cellular therapies (CAR-T and CAR-NK cells), and new platforms for allogeneic transplantation, in particular haploidentical transplants. This Special Issue, Acute Leukemias in Children: Existing Challenges and Emerging Therapies, includes contributions on some biological and treatment aspects of pediatric acute leukemia that can provide clinicians a wider overview of novel insights in the field. We hope that the present reports will be valuable for researchers and physicians investigating acute leukemia in children.

Dr. Miguel Angel Diaz
Guest Editor

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Keywords

  • pediatric acute lymphoblastic leukemia
  • pediatric acute myeloid leukemia
  • targeted therapy
  • immunotherapy
  • cellular therapy
  • allogeneic HSC transplantation
  • CAR-T cell therapy
  • CAR-NK cell therapy

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Published Papers (4 papers)

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Research

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15 pages, 1328 KiB  
Article
Osteonecrosis in Korean Paediatric and Young Adults with Acute Lymphoblastic Leukaemia or Lymphoblastic Lymphoma: A Nationwide Epidemiological Study
by Seung Min Hahn, Myeongjee Lee, Aaron Huser, Yeonji Gim, Eun Hwa Kim, Minsoo Kim, Amaal M. Aldosari, Inkyung Jung and Yoon Hae Kwak
J. Clin. Med. 2022, 11(9), 2489; https://doi.org/10.3390/jcm11092489 - 28 Apr 2022
Cited by 1 | Viewed by 1794
Abstract
Osteonecrosis (ON) is a serious complication of acute lymphocytic leukaemia (ALL) or lymphoblastic lymphoma (LBL) treatment, and there is little information regarding ON in Korean paediatric and young adult patients. This retrospective cohort study assessed the cumulative incidence of and risk factors for [...] Read more.
Osteonecrosis (ON) is a serious complication of acute lymphocytic leukaemia (ALL) or lymphoblastic lymphoma (LBL) treatment, and there is little information regarding ON in Korean paediatric and young adult patients. This retrospective cohort study assessed the cumulative incidence of and risk factors for ON using national health insurance claims data from 2008 to 2019 in 4861 ALL/LBL patients. The Kaplan–Meier method was used to estimate the cumulative incidence of ON according to age groups; the Cox proportional hazard regression model was used to identify risk factors related to ON development after diagnosing ALL/LBL. A cause-specific hazard model with time-varying covariates was used to assess the effects of risk factors. Overall, 158 (3.25%) patients were diagnosed with ON, among whom 23 underwent orthopaedic surgeries. Older age, radiotherapy (HR = 2.62, 95% confidence interval (CI) 1.87–3.66), HSCT (HR = 2.40, 95% CI 1.74–3.31), steroid use and anthracycline use (HR = 2.76, CI 1.85–4.14) were related to ON in the univariate analysis. In the multivariate analysis, age and steroid and asparaginase use (HR = 1.99, CI 1.30–3.06) were factors associated with ON. These results suggest that Korean patients with ALL/LBL who used steroids and asparaginase should be closely monitored during follow-up, even among young adult patients. Full article
(This article belongs to the Special Issue Acute Leukemias in Children: Existing Challenges and Emerging Therapies)
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10 pages, 1116 KiB  
Article
Imetelstat Induces Leukemia Stem Cell Death in Pediatric Acute Myeloid Leukemia Patient-Derived Xenografts
by Sonali P. Barwe, Fei Huang, Edward Anders Kolb and Anilkumar Gopalakrishnapillai
J. Clin. Med. 2022, 11(7), 1923; https://doi.org/10.3390/jcm11071923 - 30 Mar 2022
Cited by 9 | Viewed by 3705
Abstract
Acute myeloid leukemia (AML) in children remains deadly, despite the use of maximally intensive therapy. Because leukemia stem cells (LSCs) significantly contribute to chemoresistance and relapse, therapies that specifically target the LSCs are likely to be more beneficial in improving outcome. LSCs are [...] Read more.
Acute myeloid leukemia (AML) in children remains deadly, despite the use of maximally intensive therapy. Because leukemia stem cells (LSCs) significantly contribute to chemoresistance and relapse, therapies that specifically target the LSCs are likely to be more beneficial in improving outcome. LSCs are known to have high telomerase activity and telomerase activity is negatively correlated with survival in pediatric AML. We evaluated the preclinical efficacy of imetelstat, an oligonucleotide inhibitor of telomerase activity in patient-derived xenograft (PDX) lines of pediatric AML. Imetelstat treatment significantly increased apoptosis/death of the LSC population in a dose-dependent manner in six pediatric AML PDX lines ex vivo, while it had limited activity on the stem cell population in normal bone marrow specimens. These results were validated in vivo in two distinct PDX models wherein imetelstat as single agent or in combination with chemotherapy greatly reduced the LSC percentage and prolonged median survival. Imetelstat combination with DNA hypomethylating agent azacitidine was also beneficial in extending survival. Secondary transplantation experiments showed delayed engraftment and improved survival of mice receiving imetelstat-treated cells, confirming the diminished LSC population. Thus, our data suggest that imetelstat represents an effective therapeutic strategy for pediatric AML. Full article
(This article belongs to the Special Issue Acute Leukemias in Children: Existing Challenges and Emerging Therapies)
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9 pages, 931 KiB  
Article
Efficacy of Flotetuzumab in Combination with Cytarabine in Patient-Derived Xenograft Models of Pediatric Acute Myeloid Leukemia
by Sonali P. Barwe, Anne Kisielewski, Ezio Bonvini, John Muth, Jan Davidson-Moncada, Edward Anders Kolb and Anilkumar Gopalakrishnapillai
J. Clin. Med. 2022, 11(5), 1333; https://doi.org/10.3390/jcm11051333 - 28 Feb 2022
Cited by 5 | Viewed by 2402
Abstract
Children with acute myeloid leukemia (AML) have a poor prognosis despite the intensification of chemotherapy. Future efforts to improve outcomes should focus on more precise targeting of leukemia cells. CD123, or IL3RA, is expressed on the surface of nearly all pediatric AML samples [...] Read more.
Children with acute myeloid leukemia (AML) have a poor prognosis despite the intensification of chemotherapy. Future efforts to improve outcomes should focus on more precise targeting of leukemia cells. CD123, or IL3RA, is expressed on the surface of nearly all pediatric AML samples and is a high-priority target for immunotherapy. The efficacy of an investigational dual-affinity retargeting antibody (DART) molecule (CD123 × CD3; MGD006 or flotetuzumab) was assessed in two distinct patient-derived xenograft (PDX) models of pediatric AML. MGD006 simultaneously binds to CD123 on target cells and CD3 on effector T cells, thereby activating T cells and redirecting them to induce cytotoxicity in target cells. The concurrent treatment of cytarabine and MGD006 was performed to determine the effect of cytarabine on T-cell counts and MGD006 activity. Treatment with MGD006 along with an allogeneic human T-cell infusion to act as effector cells induced durable responses in both PDX models, with CD123 positivity. This effect was sustained in mice treated with a combination of MGD006 and cytarabine in the presence of T cells. MGD006 enhanced T-cell proliferation and decreased the burden of AML blasts in the peripheral blood with or without cytarabine treatment. These data demonstrate the efficacy of MGD006 in prolonging survival in pediatric AML PDX models in the presence of effector T cells and show that the inclusion of cytarabine in the treatment regimen does not interfere with MGD006 activity. Full article
(This article belongs to the Special Issue Acute Leukemias in Children: Existing Challenges and Emerging Therapies)
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Review

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26 pages, 843 KiB  
Review
The Development of New Agents for Post-Hematopoietic Stem Cell Transplantation Non-Infectious Complications in Children
by Uri Ilan, Erica Brivio, Mattia Algeri, Adriana Balduzzi, Marta Gonzalez-Vincent, Franco Locatelli, Christian Michel Zwaan, Andre Baruchel, Caroline Lindemans and Francisco Bautista
J. Clin. Med. 2023, 12(6), 2149; https://doi.org/10.3390/jcm12062149 - 9 Mar 2023
Cited by 3 | Viewed by 2386
Abstract
Hematopoietic stem cell transplantation (HSCT) is often the only curative treatment option for patients suffering from various types of malignant diseases and some non-cancerous conditions. Nevertheless, it is associated with a high risk of complications leading to transplant-related mortality and long-term morbidity. An [...] Read more.
Hematopoietic stem cell transplantation (HSCT) is often the only curative treatment option for patients suffering from various types of malignant diseases and some non-cancerous conditions. Nevertheless, it is associated with a high risk of complications leading to transplant-related mortality and long-term morbidity. An increasing number of therapeutic and prevention strategies have been developed over the last few years to tackle the complications arising in patients receiving an HSCT. These strategies have been mainly carried out in adults and some are now being translated into children. In this manuscript, we review the recent advancements in the development and implementation of treatment options for post-HSCT non-infectious complications in pediatric patients with leukemia and other non-malignant conditions, with a special attention on the new agents available within clinical trials. We focused on the following conditions: graft failure, prevention of relapse and early interventions after detection of minimal residual disease positivity following HSCT in acute lymphoblastic and myeloid leukemia, chronic graft versus host disease, non-infectious pulmonary complications, and complications of endothelial origin. Full article
(This article belongs to the Special Issue Acute Leukemias in Children: Existing Challenges and Emerging Therapies)
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