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Long term outcome and immune function after hematopoietic stem cell transplantation for primary immunodeficiency

Lookup NU author(s): Dr Andrew Gennery

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This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).


Abstract

© 2019 Gennery, Lankester and Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT).Transplantation techniques for patients with primary immunodeficiencies have improved so that survival from the procedure in many cases is >80%. However, long term complications may arise due to the use or not of conditioning agents. This may result 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.


Publication metadata

Author(s): Gennery AR, Lankester A

Publication type: Note

Publication status: Published

Journal: Frontiers in Pediatrics

Year: 2019

Volume: 7

Online publication date: 24/09/2019

Acceptance date: 04/09/2019

ISSN (electronic): 2296-2360

Publisher: Frontiers Media S.A.

URL: https://doi.org/10.3389/fped.2019.00381

DOI: 10.3389/fped.2019.00381


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