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SHR Neuro Cancer Cardio Lipid Metab Microb
Bakhtiar, S; Fekadu, J; Seidel, MG; Gambinieri, E.
Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Immune Dysregulatory Disorders.
Front Pediatr. 2019; 7: 461-461.
Doi: 10.3389/fped.2019.00461
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- Seidel Markus
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- Abstract:
- 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. Copyright © 2019 Bakhtiar, Fekadu, Seidel and Gambinieri.
- Find related publications in this database (Keywords)
- primary immunodeficiency
- immune dysregulation
- alloHSCT
- IPEX
- LRBA
- CTLA-4
- IL10R
- IUIS classification 2017