Cell Therapy in Pediatric Hematological Malignancies: Current Opportunities and Challenges

Dear Colleagues,

It is our great pleasure to introduce this Special Issue on “Cell Therapy in Pediatric Hematological Malignancies: Current Opportunities and Challenges”.

Research has been carried out in an attempt to improve treatment success for pediatric patients with hematological malignancies using cell therapies (hematopoietic stem cell transplantation (HSCT), donor lymphocyte infusion (DLI), and, more recently, chimeric antigen receptor (CAR-T cell) therapy). For pediatric MDS patients, allogeneic HSCT represents the only potential curative treatment. HSCT has been indicated for some pediatric patients with leukemias presenting high risk in the first complete remission. Research in HSCT has allowed improvement in outcomes post transplant, including indications for transplant, donor selection, and conditioning regimens, but further studies are necessary to avoid treatment failure mainly associated with relapse and graft-versus-host disease (GvHD). Salvage treatment options have been developed, such as donor lymphocyte infusion (DLI) and, more recently, chimeric antigen receptor (CAR-T cell) therapy. The use of CAR-T cell therapy has shown promise for the treatment of pediatric hematological malignancies, such as B-cell precursor ALL (BCP-ALL). However, there are some limitations in the use of CAR-T cells such as toxicities, antigen escape, and an immunosuppressive microenvironment. Due to the complexity of cell therapies, multidisciplinary research in basic, translational, and clinical areas are necessary to overcome these challenges.

In this Special Issue of Children, we welcome original research articles, reviews, mini-review, and case reports focusing on the current role of HSCT in pediatric hematological malignancies. Contributions may include the impact of cytogenetic and molecular alterations on outcomes, the predictive role of minimal residual disease (MRD) detection and interventions, the choice of conditioning regimens, the selection of donors, studies on GvDH, CAR-T cell therapy for pediatric hematological malignancies, CAR-T cell engineering with a discussion of the advantages and disadvantages of different approaches, strategies to improve aspects such as antigen escape, toxicities, CAR-T cell trafficking, and the immunosuppressive microenvironment.

We look forward to receiving your contributions.

Dr. Teresa de Souza Fernandez
Dr. Elaine Sobral Da Costa
Guest Editors

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• pediatric hematological malignancies
• allogeneic hematopoietic stem cell transplantation
• cytogenetic and molecular risk stratification
• donor selection
• predictive role of MRD
• conditioning regimen
• disease relapse
• GvHD
• CAR-T cell engineering approaches
• CAR-T cell and antigen escape
• immunosuppressive microenvironment