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Front Pediatr. 2019 Sep 24;7:381. doi: 10.3389/fped.2019.00381. eCollection 2019.

Long Term Outcome and Immune Function After Hematopoietic Stem Cell Transplantation for Primary Immunodeficiency.

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Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.
Paediatric Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle upon Tyne, United Kingdom.
Stem Cell Transplantation Program, Department of Pediatrics, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, Netherlands.


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.


Artemis; CGD; SCID; WAS; antibody-based conditioning; papillomavirus-associated warts; thymopoiesis

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