Universität zu Köln

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:	Creators Email ORCID ORCID Put Code Lenders, Malte UNSPECIFIED UNSPECIFIED UNSPECIFIED Schmitz, Boris UNSPECIFIED orcid.org/0000-0001-7041-7424 UNSPECIFIED Stypmann, Joerg UNSPECIFIED UNSPECIFIED UNSPECIFIED Duning, Thomas UNSPECIFIED UNSPECIFIED UNSPECIFIED Brand, Stefan-Martin UNSPECIFIED UNSPECIFIED UNSPECIFIED Kurschat, Christine UNSPECIFIED UNSPECIFIED UNSPECIFIED Brand, Eva UNSPECIFIED UNSPECIFIED UNSPECIFIED
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:	Keywords Language ALPHA-GALACTOSIDASE; CLINICAL MANIFESTATIONS; DOSE REDUCTION; SEVERITY; NEPHROPATHY; PROTEINURIA; PROGRESSION; IMPACT; COHORT; SERUM Multiple languages Transplantation; Urology & Nephrology Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/21035