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Kidney transplantation improves survival and is indicated in Fabry's disease.

Abstract：

BACKGROUND:Fabry's disease (FD) is an inborn error of glycosphingolipid catabolism with progressive systemic deposition of globotriaosylceramide thereby leading to renal and cardiac failure. Current therapy involves symptomatic medical management, dialysis, enzyme replacement therapy, kidney transplantation (KTx), and more recently gene therapy. Case fatalities occur in the fourth decade of life resulting from uremia unless dialysis or KTx is undertaken. STUDY DESIGN:This is a retrospective study aimed at determining the effect of KTx on the long-term outcome of patients with FD. RESULTS:Between 1964 and 1998, ten patients with FD received KTx at our institutions. Actuarial patient and graft survivals were 100% and 90% at 5 years; 76% and 66% at 10 years. One kidney graft was lost due to rejection. Patient survival data compared favorably at 5 years with survival of FD patients on hemodialysis alone (41%, P < .05). Five patients are alive at the time of this study, and five patients died with median survival time after KTx of 128 months (range: 74-160 months). CONCLUSIONS:This study demonstrates an excellent outcome in patients with FD in the first decade after KTx. In the absence of a severe contraindication, we advocate KTx to improve the overall prognosis of patients with renal failure due to FD. Based on the data, enzyme replacement therapy after KTx seems indicated, as FD progresses posttransplant, leading to case fatalities in the second decade after KTx.

journal_name

Transplant Proc

journal_title

Transplantation proceedings

authors

Inderbitzin D,Avital I,Largiadèr F,Vogt B,Candinas D

doi

10.1016/j.transproceed.2005.11.021

subject

Has Abstract

pub_date

2005-12-01 00:00:00

pages

4211-4

issue

10

eissn

0041-1345

issn

1873-2623

pii

S0041-1345(05)01316-3

journal_volume

37

pub_type

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