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Indications for Hematopoietic Stem Cell Transplantation in Children

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Establishing a Hematopoietic Stem Cell Transplantation Unit

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Abstract

Hematopoietic stem cell transplantation (HSCT) has significantly modified the natural history of many malignant and non-malignant disorders occurring in pediatric patients. In particular, allogeneic HSCT still represents the treatment of choice for life-threatening conditions, such as primary immune deficiencies or high-risk/relapsed leukemia, or the more effective option to improve the quality of life of patients with hemoglobinopathies. Continuous advances in transplantation outcomes achieved through the past three to four decades have led to the progressive expansion of the indications for HSCT, which is now used with success in several malignant and non-malignant disorders.

Since 2010, more than 30,000 transplants have been reported each year to the European Group for Blood and Marrow Transplantation (EBMT), with a record number of 40,829 HSCTs, in 36,469 patients, reported for the 2014 EBMT activity survey (16,946 allogeneic and 23,883 autologous HSCTs). Of the 656 currently active institutions, 107 (16%) are dedicated pediatric transplant centers and 118 centers (18%) perform HSCTs in both adults and children. These findings indicate that transplantation programs reserved for children must be implemented for those centers willing to take care of the pediatric population of patients. The number of HSCTs increases every year, with a continuous expansion of the use of human leucocyte antigen (HLA)-haploidentical transplantation, a slower increment for unrelated donor HSCTs, and a progressive reduction in cord blood use.

The main indications for allogeneic HSCT in the pediatric age group reported to the EBMT during the 2012 activity survey included acute lymphoblastic leukemia (26%) and primary immune-deficiencies (16%), while solid tumors (66%, in particular neuroblastoma) and lymphomas (15%) were the main indications for autologous HSCT.

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Authors and Affiliations

  1. Department of Pediatric Hematology/Oncology, IRCCS, Bambino Gesù Children’s Hospital, Piazza Sant’Onofrio, 4, Rome, 00165, Italy

    Franco Locatelli M.D., Ph.D.

  2. Department of Pediatric Science, University of Pavia, Pavia, 27100, Italy

    Franco Locatelli M.D., Ph.D. & Luisa Strocchio M.D.

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  1. Franco Locatelli M.D., Ph.D.
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  2. Luisa Strocchio M.D.
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Correspondence to Franco Locatelli M.D., Ph.D. .

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Editors and Affiliations

  1. Eurocord Hôpital Saint Louis, Paris, France

    Éliane Gluckman

  2. Department of Hematology and Medical Oncology, University of Leipzig, Leipzig, Germany

    Dietger Niederwieser

  3. Oncology Center, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia

    Mahmoud Aljurf

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Locatelli, F., Strocchio, L. (2018). Indications for Hematopoietic Stem Cell Transplantation in Children. In: Gluckman, É., Niederwieser, D., Aljurf, M. (eds) Establishing a Hematopoietic Stem Cell Transplantation Unit . Springer, Cham. https://doi.org/10.1007/978-3-319-59358-6_6

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