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Long-Term Outcomes of Kidney Transplantation in Fabry Disease
- Source :
- Ersözlü, Sara; Desnick, Robert J; Huynh-Do, Uyen; Canaan-Kühl, Sima; Barbey, Frédéric; Genitsch, Vera; Mueller, Thomas F; Cheetham, Marcus; Flammer, Andreas J; Schaub, Stefan; Nowak, Albina (2018). Long-term Outcomes of Kidney Transplantation in Fabry Disease. Transplantation, 102(11), pp. 1924-1933. Wolters Kluwer Health 10.1097/TP.0000000000002252
- Publication Year :
- 2018
-
Abstract
- BACKGROUND 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. METHODS 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. RESULTS 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. CONCLUSIONS We conclude that kidney transplantation has an excellent long-term outcome in FD.
- Subjects :
- Male
Time Factors
2747 Transplantation
Endocrinology, Diabetes and Metabolism
030232 urology & nephrology
Disease
030204 cardiovascular system & hematology
Biochemistry
0302 clinical medicine
Endocrinology
Germany
Long term outcomes
10035 Clinic for Nephrology
610 Medicine & health
Kidney transplantation
Kidney
Graft Survival
Middle Aged
Treatment Outcome
medicine.anatomical_structure
Disease Progression
10209 Clinic for Cardiology
Female
Kidney Diseases
Switzerland
Adult
medicine.medical_specialty
Adolescent
Urology
Nephropathy
Young Adult
03 medical and health sciences
medicine
Genetics
Humans
Enzyme Replacement Therapy
Molecular Biology
Transplantation
business.industry
Patient survival
medicine.disease
Kidney Transplantation
Fabry disease
Fabry Disease
Kidney Failure, Chronic
570 Life sciences
biology
10029 Clinic and Policlinic for Internal Medicine
business
Subjects
Details
- Language :
- English
- Database :
- OpenAIRE
- Journal :
- Ersözlü, Sara; Desnick, Robert J; Huynh-Do, Uyen; Canaan-Kühl, Sima; Barbey, Frédéric; Genitsch, Vera; Mueller, Thomas F; Cheetham, Marcus; Flammer, Andreas J; Schaub, Stefan; Nowak, Albina (2018). Long-term Outcomes of Kidney Transplantation in Fabry Disease. Transplantation, 102(11), pp. 1924-1933. Wolters Kluwer Health 10.1097/TP.0000000000002252 <http://dx.doi.org/10.1097/TP.0000000000002252>
- Accession number :
- edsair.doi.dedup.....e328931fa58a2b9a65d16ef8ca0a1f64