Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Immune Dysregulatory Disorders.

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 to

SUBMITTER: Bakhtiar S 

PROVIDER: S-EPMC6865355 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature