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Haploidentical Stem Cell Transplantation pp 149–178Cite as

Applications of Haploidentical SCT in Pediatric Patients

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Part of the book series: Stem Cell Biology and Regenerative Medicine ((STEMCELL))

Abstract

Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is increasingly used as potentially curative option for pediatric patients affected by leukemia or other malignancies, as well as by several non malignant disorders such as primary immunodeficiencies, constitutional and acquired bone marrow failure syndromes, hemoglobinopathies and others.

Different techniques have been developed in order to overcome the HLA barrier and make HSCT feasible in virtually all patients. These approaches are mainly based on the in vitro physical removal of alloreactive T cells capable of mediating graft-versus-host disease (GVHD) or on in vivo pharmacological treatment with immunosuppressive drugs or immunomodulating agents.

The possibility to receive a transplant from a haploidentical donor (usually a parent) is particularly relevant both for children needing to have rapid access to this procedure, and for patients belonging to ethnic minorities or with rare HLA haplotypes that are underrepresented in the international voluntary donor registries.

The delayed immune reconstitution after haplo-HSCT may cause impaired graft-versus-leukemia (GVL) effect and defective control of potentially life-threatening opportunistic infections, thus increasing the risk of relapse and of transplant-related mortality (TRM). For this reason, several strategies of post-transplant adoptive cell therapy have been developed over time, from the use of donor lymphocyte infusions (DLI), to the administration of selected immunocompetent cell populations or genetically modified T lymphocytes, or the production of leukemia or pathogen-specific cytotoxic T cells (CTL) to be infused after the transplant.

Even if prospective clinical trials are still needed in order to define the optimal strategy to treat each different disease, the results of haplo-HSCT in children have significantly improved over time and, nowadays, the probability of disease cure is often similar to that of transplants performed from matched unrelated donors, thus making this approach extremely appealing in the pediatric population.

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Authors and Affiliations

  1. Pediatric Hematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

    Marco Zecca

  2. Cell Factory GMP Laboratory, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

    Patrizia Comoli

Authors
  1. Marco Zecca
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  2. Patrizia Comoli
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Corresponding author

Correspondence to Marco Zecca .

Editor information

Editors and Affiliations

  1. Department of Hematology, Ankara University Medical School, Sıhhiye, Ankara, Turkey

    Taner Demirer

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Zecca, M., Comoli, P. (2017). Applications of Haploidentical SCT in Pediatric Patients. In: Demirer, T. (eds) Haploidentical Stem Cell Transplantation. Stem Cell Biology and Regenerative Medicine. Humana Press, Cham. https://doi.org/10.1007/978-3-319-65319-8_11

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