Allogeneic hematopoietic stem cell transplantation for congenital immune dysregulatory disorders

Primary immunodeficiency disorders that predominantly affect immune regulation and mechanisms of self-tolerance have come into the limelight, because at least for a subgroup of monogenetic disorders, a targeted therapy h
Primary immunodeficiency disorders that predominantly affect immune regulation and mechanisms of self-tolerance have come into the limelight, because at least for a subgroup of monogenetic disorders, a targeted therapy has become available. Nevertheless, their management often involves the treatment of severely compromising, refractory, multi-organ autoimmunity, leading to further increased susceptibility to infections and complications of long-term immune suppressive treatment, including the risk of malignancy. While evidence for allogeneic hematopoietic stem cell transplantation (alloHSCT) as a curative treatment option for severely affected patients by this disease category accumulates, clear indications, and guidelines for alloHSCT are lacking. Predictive and stratification-relevant tools such as disease activity scores are largely missing and often there is not a consistent genotype-phenotype correlation within the same family to facilitate the decision whether to transplant or not. In this review, we provide a literature-based update on indications and outcomes of alloHSCT for congenital immune dysregulative inborn errors of immunity according to the IUIS classification 2017.
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Author:Shahrzad Bakhtiar, Julia Fekadu, Markus G. Seidel, Eleonora Gambinieri
URN:urn:nbn:de:hebis:30:3-518862
DOI:http://dx.doi.org/10.3389/fped.2019.00461
ISSN:2296-2360
Pubmed Id:http://www.ncbi.nlm.nih.gov/pubmed?term=31799221
Parent Title (English):Frontiers in Pediatrics
Publisher:Frontiers Media
Place of publication:Lausanne
Contributor(s):Arjan C. Lankester
Document Type:Article
Language:English
Year of Completion:2019
Date of first Publication:2019/11/13
Publishing Institution:Universitätsbibliothek Johann Christian Senckenberg
Contributing Corporation:Inborn Errors Working Party Group of the EBMT
Release Date:2019/12/18
Tag:CTLA-4; IL10R; IPEX; IUIS classification 2017; LRBA; alloHSCT; immune dysregulation; primary immunodeficiency
Volume:7
Issue:Art. 461
Pagenumber:10
First Page:1
Last Page:10
Note:
Copyright © 2019 Bakhtiar, Fekadu, Seidel and Gambinieri. 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.
HeBIS PPN:458140988
Institutes:Medizin
Dewey Decimal Classification:610 Medizin und Gesundheit
Sammlungen:Universitätspublikationen
Licence (German):License LogoCreative Commons - Namensnennung 4.0

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