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Haploidentical Transplants for Nonmalignant Diseases in Children

  • Christian Seitz
  • Patrick Schlegel
  • Rupert Handgretinger
Chapter
Part of the Advances and Controversies in Hematopoietic Transplantation and Cell Therapy book series (ACHTCT)

Abstract

Allogeneic hematopoietic cell transplantation (allo-HCT) offers a curative treatment strategy to a variety of nonmalignant hematological and immunological disorders as well as inborn errors of metabolism. Successful allo-HCT is able to restore functional hematopoiesis and immune function and can substitute disabled enzymatic activity. Nevertheless, allo-HCT can also be associated with serious risks for transplantation-related morbidities or even mortalities like graft-versus-host disease (GvHD) or life-threatening infectious complications. Especially in nonmalignant disorders (NMD), risks and benefits have to be carefully balanced on an individual patient basis. Up to now, human leukocyte antigen (HLA)-matched siblings are the preferred source of hematopoietic graft. Only about one third of patients have HLA-matched sibling donor (MSD) and the number further decreases in patients with inherited disorders as siblings might be carriers or affected as well. HLA-matched unrelated donors (MUD) have become an important alternative. Chances of finding a HLA-MUD are particularly dismal for individuals belonging to certain ethnic groups, with often less than 10% compared to approximately 75% in the Caucasian population. Therefore, alternative donor sources have to been taken into account, especially when the clinical condition of the patients does not allow a further delay of the allo-HCT. This chapter focuses on HLA-haploidentical hematopoietic cell transplant (haplo-HCT) in NMD. It will highlight recent developments in graft manipulation utilizing safe application of haplo-HCT grafts and discuss important advantages which might lift haplo-HCT to a standard therapy in NMDs in the near future.

Keywords

Haploidentical transplant GvHD Nonmalignant disorders NK-cells T-cell depletion Bone marrow failure syndromes Fanconi anemia SCID Inborn errors Metabolism Immunodeficiency Thalassemia 

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Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  • Christian Seitz
    • 1
  • Patrick Schlegel
    • 1
  • Rupert Handgretinger
    • 1
  1. 1.Department of Hematology/OncologyChildren’s University HospitalTübingenGermany

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