Long-term Outcomes of Kidney Transplantation in Fabry Disease.

Details

Serval ID
serval:BIB_A9CE7896FB30
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Long-term Outcomes of Kidney Transplantation in Fabry Disease.
Journal
Transplantation
Author(s)
Ersözlü S., Desnick R.J., Huynh-Do U., Canaan-Kühl S., Barbey F., Genitsch V., Mueller T.F., Cheetham M., Flammer A.J., Schaub S., Nowak A.
ISSN
1534-6080 (Electronic)
ISSN-L
0041-1337
Publication state
Published
Issued date
11/2018
Peer-reviewed
Oui
Volume
102
Number
11
Pages
1924-1933
Language
english
Notes
Publication types: Journal Article ; Multicenter Study
Publication Status: ppublish
Abstract
Fabry disease (FD) is a rare X-linked lysosomal storage disorder caused by mutations in the α-galactosidase A gene that obliterate or markedly reduce α-galactosidase A activity. This results in the systemic accumulation of its glycosphingolipid substrates in body fluids and organs, including the kidney. Fabry nephropathy can lead to end-stage renal disease requiring kidney transplantation. Little is known about its long-term outcomes and the overall patient survival after kidney transplantation.
Here, we report 17 Fabry patients (15 male and 2 female subjects) who received kidney transplants and their long-term treatment and follow-up at 4 specialized Fabry centers.
The posttransplant follow-up ranged to 25 years, with a median of 11.5 (range, 0.8-25.5] years. Graft survival was similar, and death-censored graft survival was superior to matched controls. Fabry patients died with functioning kidneys, mostly from cardiac causes. In 2 male subjects 14 and 23 years posttransplant, the grafts had a few typical FD lamellar inclusions, presumably originating from invading host macrophages and vascular endothelial cells.
We conclude that kidney transplantation has an excellent long-term outcome in FD.
Keywords
Adolescent, Adult, Disease Progression, Enzyme Replacement Therapy, Fabry Disease/complications, Fabry Disease/drug therapy, Fabry Disease/genetics, Fabry Disease/mortality, Female, Germany, Graft Survival, Humans, Kidney Diseases/etiology, Kidney Diseases/mortality, Kidney Diseases/surgery, Kidney Failure, Chronic/etiology, Kidney Failure, Chronic/surgery, Kidney Transplantation/adverse effects, Kidney Transplantation/mortality, Male, Middle Aged, Switzerland, Time Factors, Treatment Outcome, Young Adult
Pubmed
Web of science
Create date
26/04/2018 16:51
Last modification date
15/01/2020 6:26
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