Lenders, Malte, Schmitz, Boris ORCID: 0000-0001-7041-7424, Stypmann, Joerg, Duning, Thomas, Brand, Stefan-Martin, Kurschat, Christine and Brand, Eva (2017). Renal function predicts long-term outcome on enzyme replacement therapy in patients with Fabry disease. Nephrol. Dial. Transplant., 32 (12). S. 2090 - 2098. OXFORD: OXFORD UNIV PRESS. ISSN 1460-2385

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Abstract

Background. Renal and cardiac involvement is responsible for substantial morbidity and mortality in Fabry disease (FD). We analysed the incidence of FD-related renal, cardiac and neurologic end points in patients with FD on long-term enzyme replacement therapy (ERT). Methods. A retrospective analysis of prospectively collected data from two German FD centres was performed. The impact of renal and cardiac function at ERT-naive baseline on end point development despite ERT was analysed. Results. Fifty-four patients (28 females) receiving ERT (mean 81 +/- 21 months) were investigated. Forty per cent of patients were diagnosed with clinical end points before ERT initiation and 50% of patients on ERT developed new clinical end points. In patients initially diagnosed with an end point before ERT initiation, the risk for an additional end point on ERT was increased fhazard ratio [HR] 3.83 [95% confidence interval (CI) 1.61-9.08]; P = 0.0023g. A decreased glomerular filtration rate (eGFR) <= 75 mL/min/1.73 m(2) in ERT-naive patients at baseline was associated with an increased risk for cardiovascular end points [HR 3.59 (95% CI 1.15-11.18); P = 0.0273] as well as for combined renal, cardiac and neurologic end points on ERT [HR 4.77 (95% CI 1.93-11.81); P = 0.0007]. In patients with normal kidney function, left ventricular hypertrophy at baseline predicted a decreased end point-free survival [HR 6.90 (95% CI 2.04-23.27); P = 0.0018]. The risk to develop an end point was independent of sex. Conclusions. In addition to age, even moderately impaired renal function determines FD progression on ERT. In patients with FD, renal and cardiac protection is warranted to prevent patients from deleteriousmanifestations of the disease.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Lenders, MalteUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Schmitz, BorisUNSPECIFIEDorcid.org/0000-0001-7041-7424UNSPECIFIED
Stypmann, JoergUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Duning, ThomasUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Brand, Stefan-MartinUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Kurschat, ChristineUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Brand, EvaUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-210351
DOI: 10.1093/ndt/gfw334
Journal or Publication Title: Nephrol. Dial. Transplant.
Volume: 32
Number: 12
Page Range: S. 2090 - 2098
Date: 2017
Publisher: OXFORD UNIV PRESS
Place of Publication: OXFORD
ISSN: 1460-2385
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
ALPHA-GALACTOSIDASE; CLINICAL MANIFESTATIONS; DOSE REDUCTION; SEVERITY; NEPHROPATHY; PROTEINURIA; PROGRESSION; IMPACT; COHORT; SERUMMultiple languages
Transplantation; Urology & NephrologyMultiple languages
Refereed: Yes
URI: http://kups.ub.uni-koeln.de/id/eprint/21035

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