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Advances in Pediatric Cancer Therapy

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

Deadline for manuscript submissions: closed (30 November 2022) | Viewed by 6250

Special Issue Editors


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Guest Editor
Department of Paediatric Haematology/Oncology, Cell and Gene Therapy, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy
Interests: malignant hematology; lymphoma; leukemia
Special Issues, Collections and Topics in MDPI journals

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Guest Editor
Department of Paediatric Haematology/Oncology, Cell and Gene Therapy, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy
Interests: neuroblastoma; retinoblastoma; COVID-19; drug therapy; pediatric
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Since the introduction of chemotherapy, much progress has been made and a dramatic improvement has been seen in the last 50 years, with an actual cure rate of childhood cancer of 80% compared to < 25% in the pre-chemotherapy era.

Progress in pediatric hematology and oncology is mostly correlated to the identification of clinical and biologic prognostic factors, leading to risk-adapted therapeutic approaches with treatment intensity modulated to the molecular characteristics at diagnosis as well as to the response in the course of treatment. The risk of late effects associated with more intensive treatment (including high-dose chemotherapy and radiotherapy) are widely recognized nowadays. Therefore, new therapeutic approaches combining less intensive chemotherapy and innovative treatments are under investigation with the aim to increase overall survival and minimize late mortality and side effects, including cardiac deaths, infertility and second cancers. Advances in the understanding of the genetics of childhood cancer have allowed the identification of molecular targets that can potentially be exploited for therapeutic benefits. A wide variety of novel agents that target specific genetic lesions (i.e., bevacizumab, bortezomib, vorinostat, sorafenib and mTOR inhibitors) are under evaluation in subgroups of relapsed/refractory patients in both prospective clinical trials and retrospective series. Other therapeutic approaches characterized by immunotherapeutic strategies, such as the use of monoclonal antibodies, bispecific T-cell engagers (BiTEs) and the use of chimeric antigen receptor (CAR) T-cells, are improving remission rates in refractory/relapsed disease, principally in the field of hematologic malignancies, and are under evaluation in first-line settings.

Dr. Luciana Vinti
Dr. Maria Antonietta De Ioris
Guest Editors

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Keywords

  • pediatric
  • cancer
  • immunotherapy
  • novel agents
  • target therapy
  • pediatric hematologic malignancy
  • pediatric oncology
  • hematopoietic stem cell transplantation
  • solid tumor
  • acute leukemia
  • lymphoma

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Published Papers (2 papers)

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Research

15 pages, 977 KiB  
Article
The Importance of New EBMT Criteria on the Diagnosis of Veno-Occlusive Liver Disease in Children
by Mária Füssiová, Peter Švec, Júlia Horáková, Petr Sedláček, Peter Rohoň, Peter Celec, Ivana Boďová, Jaroslava Adamčáková, Tomáš Sýkora, Veronika Dobšinská, Miroslava Pozdechová, Dominika Dóczyová, Santia Vargová and Alexandra Kolenová
J. Clin. Med. 2023, 12(3), 826; https://doi.org/10.3390/jcm12030826 - 20 Jan 2023
Cited by 3 | Viewed by 2903
Abstract
Background: Early recognition and specific therapy facilitate a favorable disease course in hepatic venous-occlusive disease (HVOD) following hematopoietic stem cell transplantation (HCT). Diagnostic and classification criteria, published by the European Society for Blood and Marrow Transplantation (EBMT), better account for clinical differences in [...] Read more.
Background: Early recognition and specific therapy facilitate a favorable disease course in hepatic venous-occlusive disease (HVOD) following hematopoietic stem cell transplantation (HCT). Diagnostic and classification criteria, published by the European Society for Blood and Marrow Transplantation (EBMT), better account for clinical differences in disease presentation in pediatric populations. Objectives: To compare the course of HVOD in children before and after the implementation of new EBMT criteria. Material and methods: The study retrospectively evaluates 26 HVODs in 179 children treated in a single HCT unit (Slovakia) comparing the period of 2014–2017 using the Baltimore and modified Seattle criteria with the period of 2018–2021, when new EBMT criteria were adopted. Results: No difference in HVOD incidence (11.2% vs. 14.8%, p = 0.46) and in time of diagnosis post-HCT (15.6 days vs. 15.7 days, p = 0.75) was found. With EBMT criteria we observed more frequent anicteric disease at diagnosis (50% vs. 87.5%, p = 0.04), lower serum bilirubin at diagnosis (3.4 mg/dL vs. 1.23 mg/dL, p = 0.045), and non-significant trends of shorter defibrotide treatment (21.7 days vs. 15.6 days, p = 0.73), decreased mortality (30% vs. 6.2%, p = 0.10) and shorter hospitalization (73.1 days vs. 59.6 days, p = 0.54). Conclusions: Different time periods around the implementation of new criteria are evaluated, underling that pediatric EBMT criteria for post-transplant HVOD diagnosis appear more sensitive. Full article
(This article belongs to the Special Issue Advances in Pediatric Cancer Therapy)
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13 pages, 2143 KiB  
Article
Pre-Transplant Total Lymphocyte Count Determines Anti-Thymocyte Globulin Exposure, Modifying Graft-versus-Host Disease Incidence and Post-Transplant Thymic Restoration: A Single-Center Retrospective Study
by Antonio Giacomo Grasso, Roberto Simeone, Alessandra Maestro, Davide Zanon and Natalia Maximova
J. Clin. Med. 2023, 12(2), 730; https://doi.org/10.3390/jcm12020730 - 16 Jan 2023
Cited by 2 | Viewed by 2642
Abstract
The use of anti-thymocyte globulin (ATG) as part of conditioning to prevent graft-versus-host disease (GVHD) may severely impair immune reconstitution (IR). We analyzed relationships between ATG exposure, the recipient lymphocyte count, IR, and transplant outcome. We retrospectively reviewed patients aged ≤ 18 years [...] Read more.
The use of anti-thymocyte globulin (ATG) as part of conditioning to prevent graft-versus-host disease (GVHD) may severely impair immune reconstitution (IR). We analyzed relationships between ATG exposure, the recipient lymphocyte count, IR, and transplant outcome. We retrospectively reviewed patients aged ≤ 18 years who underwent allogeneic HSCT between April 2005 and April 2020. The outcomes of interest included the incidence of GVHD, overall survival (OS), and IR. IR was analyzed through thymic magnetic resonance imaging (MRI) and by quantifying T CD4+ and recent thymic emigrants (RTEs). The ATG-exposed group was split into a low ATG/lymphocyte ratio subgroup (ratio < 0.01) and a high ATG/lymphocyte ratio subgroup (ratio > 0.01). The low ratio subgroup had a higher incidence of GVHD (29 [59%] vs. 7 [16.6%]) but a better IR in both laboratory and MRI imaging assessments (p < 0.0001). The median thymic volume in the low ratio subgroup was significantly higher (14.7 cm3 vs. 4.5 cm3, p < 0.001). This was associated with a better OS and lower transplant-related mortality (TRM) (80.4% vs. 58.0%, p = 0.031) and (13.1% vs. 33.0%, p = 0.035). An individualized approach to ATG dosing allows for the obtainment of rapid thymic reconstitution and the best transplant-related outcomes. Full article
(This article belongs to the Special Issue Advances in Pediatric Cancer Therapy)
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