{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,4]],"date-time":"2026-04-04T08:07:19Z","timestamp":1775290039531,"version":"3.50.1"},"reference-count":36,"publisher":"Oxford University Press (OUP)","issue":"12","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2017,12,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>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).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A retrospective analysis of prospectively collected data from two German FD centres was performed. The impact of renal and cardiac function at ERT-na\u00efve baseline on end point development despite ERT was analysed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Fifty-four patients (28 females) receiving ERT (mean 81 \u00b1 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 {hazard ratio [HR] 3.83 [95% confidence interval (CI) 1.61\u20139.08]; P = 0.0023}. A decreased glomerular filtration rate (eGFR) \u226475 mL\/min\/1.73 m2 in ERT-na\u00efve patients at baseline was associated with an increased risk for cardiovascular end points [HR 3.59 (95% CI 1.15\u201311.18); P = 0.0273] as well as for combined renal, cardiac and neurologic end points on ERT [HR 4.77 (95% CI 1.93\u201311.81); P = 0.0007]. In patients with normal kidney function, left ventricular hypertrophy at baseline predicted a decreased end point\u2013free survival [HR 6.90 (95% CI 2.04\u201323.27); P = 0.0018]. The risk to develop an end point was independent of sex.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>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 deleterious manifestations of the disease.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1093\/ndt\/gfw334","type":"journal-article","created":{"date-parts":[[2016,9,27]],"date-time":"2016-09-27T20:58:29Z","timestamp":1475009909000},"page":"2090-2097","source":"Crossref","is-referenced-by-count":10,"title":["Renal function predicts long-term outcome on enzyme replacement therapy in patients with Fabry disease"],"prefix":"10.1093","volume":"32","author":[{"given":"Malte","family":"Lenders","sequence":"first","affiliation":[{"name":"University Hospital Muenster Department of Nephrology, Hypertension, and Rheumatology, Internal Medicine D , , Albert-Schweitzer-Campus 1, D-48149 Muenster ,","place":["Germany"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Boris","family":"Schmitz","sequence":"additional","affiliation":[{"name":"University Hospital Muenster Institute of Sports Medicine, Molecular Genetics of Cardiovascular Disease , , Muenster ,","place":["Germany"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J\u00f6rg","family":"Stypmann","sequence":"additional","affiliation":[{"name":"University Hospital Muenster Department of Cardiovascular Medicine, Division of Cardiology , , Muenster ,","place":["Germany"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Thomas","family":"Duning","sequence":"additional","affiliation":[{"name":"University Hospital Muenster Department of Neurology , , Muenster ,","place":["Germany"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stefan-Martin","family":"Brand","sequence":"additional","affiliation":[{"name":"University Hospital Muenster Institute of Sports Medicine, Molecular Genetics of Cardiovascular Disease , , Muenster ,","place":["Germany"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christine","family":"Kurschat","sequence":"additional","affiliation":[{"name":"University of Cologne Department II of Internal Medicine and Center for Molecular Medicine Cologne , , Cologne ,","place":["Germany"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eva","family":"Brand","sequence":"additional","affiliation":[{"name":"University Hospital Muenster Department of Nephrology, Hypertension, and Rheumatology, Internal Medicine D , , Albert-Schweitzer-Campus 1, D-48149 Muenster ,","place":["Germany"]}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"286","published-online":{"date-parts":[[2016,9,27]]},"reference":[{"key":"2025102312501451200_GFW334C1","doi-asserted-by":"crossref","first-page":"2771","DOI":"10.1001\/jama.284.21.2771","article-title":"Clinical features of and recent advances in therapy for Fabry 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