Saving the red baby successful allogeneic cord blood transplantation in Omenn syndrome

Saving the red baby : successful allogeneic cord blood transplantation in Omenn syndrome

Haematopoietic stem cell transplantation is the treatment of choice for severe primary immunodeficiencies, but only has moderate prognosis in Omenn syndrome as it is complicated by highly activated Omenn T-cells resulting in delayed T-cell engraftment and a high rate of graft failure. A 6 1/2 months...

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Veröffentlicht in:Clinical immunology (Orlando, Fla.). - 1999. - 130(2009), 3 vom: 15. März, Seite 259-63
1. Verfasser: Schönberger, Stefan (VerfasserIn)
Weitere Verfasser: Ott, Hagen, Gudowius, Sonja, Wüller, Stefan, Baron, Jens Malte, Merk, Hans-Friedrich, Lassay, Lisa, Megahed, Mosaad, Feyen, Oliver, Laws, Hans-Jürgen, Dilloo, Dagmar, Borkhardt, Arndt, Niehues, Tim
Format: Online-Aufsatz
Sprache:English
Veröffentlicht: 2009
Zugriff auf das übergeordnete Werk:Clinical immunology (Orlando, Fla.)
Schlagworte:Case Reports Journal Article
Beschreibung
Zusammenfassung:Haematopoietic stem cell transplantation is the treatment of choice for severe primary immunodeficiencies, but only has moderate prognosis in Omenn syndrome as it is complicated by highly activated Omenn T-cells resulting in delayed T-cell engraftment and a high rate of graft failure. A 6 1/2 months old patient with a previously unknown compound heterozygous defect within the RAG1 gene (R474C; R975W) underwent 8/10 HLA-matched cord blood transplantation after myeloablative conditioning. Immune reconstitution was impressive with T-, B- and NK-cells reaching the median of age-dependent reference values within twelve, four and two months respectively. With a continuous decrease of activated Omenn T-cells there was a steady increase of naive, probably thymus-derived T-cells. Polyclonal B-cell activation and hypergammaglobulinaemia disappeared with B-cell engraftment. This case emphasizes that, despite their naive status and HLA-barriers, cord blood T-cells were apparently able to achieve T-effector function resulting in the elimination of all activated Omenn T-cells
Beschreibung:Date Completed 03.03.2009
Date Revised 09.02.2009
published: Print-Electronic
Citation Status MEDLINE
ISSN:1521-7035
DOI:10.1016/j.clim.2008.09.018