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World J Transplant. Jul 31, 2019; 9(3): 48-57
Published online Jul 31, 2019. doi: 10.5500/wjt.v9.i3.48
Considerations for hematopoietic stem cell transplantation in primary immunodeficiency disorders
Tatyana Gavrilova
Tatyana Gavrilova, Division of Allergy and Immunology, Montefiore Medical Center, Bronx, NY 10461, United States
Author contributions: Tatyana Gavrilova is the sole contributor to this manuscript and is solely responsible for its content.
Conflict-of-interest statement: The authors declare that they have no competing interests.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Corresponding author: Tatyana Gavrilova, MD, Assistant Professor, Division of Allergy and Immunology, Montefiore Medical Center; Albert Einstein College of Medicine, 1525 Blondell Avenue, Bronx, NY 10461, United States. tgavrilo@montefiore.org
Telephone: +1-718-405-8530
Received: March 21, 2019
Peer-review started: March 24, 2019
First decision: April 11, 2019
Revised: June 10, 2019
Accepted: July 16, 2019
Article in press: July 16, 2019
Published online: July 31, 2019
Abstract

Primary immunodeficiency disorders (PIDs) result from inborn errors in immunity. Susceptibility to infections and oftentimes severe autoimmunity pose life-threatening risks to patients with these disorders. Hematopoietic cell transplant (HCT) remains the only curative option for many. Severe combined immunodeficiency disorders (SCID) most commonly present at the time of birth and typically require emergent HCT in the first few weeks of life. HCT poses an unusual challenge for PIDs. Donor source and conditioning regimen often impact the outcome of immune reconstitution after HCT in PIDs. The use of matched or unmatched, as well as related versus unrelated donor has resulted in variable outcomes for different subsets of PIDs. Additionally, there is significant variability in the success of engraftment even for a single patient’s lymphocyte subpopulations. While certain cell lines do well without a conditioning regimen, others will not reconstitute unless conditioning is used. The decision to proceed with a conditioning regimen in an already immunocompromised host is further complicated by the fact that alkylating agents should be avoided in radiosensitive PIDs. This manuscript reviews some of the unique elements of HCT in PIDs and evidence-based approaches to transplant in patients with these rare and challenging disorders.

Keywords: Primary immunodeficiency disorders, Hematopoietic stem cell transplant, Autoimmunity, Conditioning regimens, Engraftment

Core tip: Primary immunodeficiency disorders (PIDs) result from inborn errors in immunity and hematopoietic cell transplant (HCT) still remains the only curative option for many of these disorders. Severe combined immunodeficiency disorders are a medical emergency and require HCT within the first few weeks of life. Optimal donor selection, conditioning regimen and outcomes of immune reconstitution vary greatly among these disorders. This manuscript reviews some of the unique elements of HCT in PIDs and evidence-based approaches to transplant in patients with these rare and complex disorders.