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Front. Pediatr. | doi: 10.3389/fped.2019.00381

Long term outcome and immune function after Haematopoietic Stem Cell Transplantation for Primary Immunodeficiency

  • 1Institute of Cellular Medicine, Newcastle University, United Kingdom
  • 2Great North Children's Hospital, United Kingdom
  • 3Willem-Alexander Children's Hospital, Leiden University Medical Center, Netherlands

Transplantation techniques for patients with primary immunodeficiencies have improved so that survival from the procedure in many cases is around 90%. However, long term complications may arise as a result of the use of conditioning agents or not, resulting in variable immune reconstitution, the long term effects of chemotherapy, particularly on fertility, and complications relating to the genetic disorder, unresolved by transplantation.
For patients with severe combined immunodeficiency (SCID), long term T- and B-lymphocyte immune reconstitution is best achieved after pre-transplant chemotherapy. For patients who receive an unconditioned infusion of donor stem cells, the quality of immune reconstitution depends on the SCID genotype. Long term effects include chemotherapy-induced impaired fertility, and sequelae specific to the genotype.
For patients with other primary immunodeficiencies, conditioning is required – sequelae related to direct effects of chemotherapy may be observed. Additional long term effects may be observed due to partial donor chimerism resulting in incomplete eradication of disease, and other geno-specific effects.

Keywords: Severe Combined Immunodeficiency, thymopoiesis;, ADA-deficiency, ARTEMIS, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease;

Received: 10 Jul 2019; Accepted: 04 Sep 2019.

Copyright: © 2019 Gennery and Lankester. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: Prof. Andrew R. Gennery, Newcastle University, Institute of Cellular Medicine, Newcastle upon Tyne, NE1 4LP, United Kingdom,