{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T16:33:49Z","timestamp":1793205229780,"version":"4.5.0"},"reference-count":31,"publisher":"Oxford University Press (OUP)","issue":"3","license":[{"start":{"date-parts":[[2025,12,19]],"date-time":"2025-12-19T00:00:00Z","timestamp":1766102400000},"content-version":"vor","delay-in-days":2123,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2020,2,26]],"date-time":"2020-02-26T00:00:00Z","timestamp":1582675200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013995","name":"Sanofi Genzyme","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013995","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100013995","name":"Sanofi Genzyme","doi-asserted-by":"publisher","award":["\u2010"],"award-info":[{"award-number":["\u2010"]}],"id":[{"id":"10.13039\/100013995","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2020,6,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Aims<\/jats:title>\n                    <jats:p>Long-term treatment effect studies in large female Fabry patient groups are challenging to design because of phenotype heterogeneity and lack of appropriate comparison groups, and have not been reported. We compared long-term cardiomyopathy and kidney function outcomes after agalsidase beta treatment with preceding treatment-naive outcomes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods and results<\/jats:title>\n                    <jats:p>Self-controlled pretreatment and post-treatment comparison (piecewise mixed linear modelling) included Fabry female patients \u226518 years at treatment initiation who received agalsidase beta (0.9\u20131.1 mg\/kg every other week) for \u22652 years, with \u22652 pretreatment and \u22652 post-treatment outcome measurements during 10-year follow-up. Left ventricular posterior wall thickness (LVPWT)\/interventricular septal thickness (IVST) and estimated glomerular filtration rate (eGFR, Chronic Kidney Disease Epidemiology Collaboration creatinine equation) analyses included 42 and 86 patients, respectively, aged 50.0 and 46.3 years at treatment initiation, respectively. LVPWT and IVST increased pretreatment (follow-up 3.5 years) but stabilized during 3.6 years of treatment (LVPWT: n = 38, slope difference [95% confidence interval (CI)] = \u22120.41 [\u22120.68, \u22120.15] mm\/year, P \u00a0pre\u2013post difference &amp;lt;0.01; IVST: n = 38, slope difference = \u22120.32 [\u22120.67, 0.02] mm\/year, P \u00a0pre\u2013post difference = 0.07). These findings were not modified by renal involvement or antiproteinuric agent use. Compared with the treatment-naive period (follow-up 3.6 years), eGFR decline remained modest and stabilized within normal ranges during 4.1 years of treatment (slope difference, 95% CI: \u22120.13 [\u22121.15, 0.89] mL\/min\/1.73m2\/year, P \u00a0pre\u2013post difference = 0.80).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Cardiac hypertrophy, progressing during pretreatment follow-up, appeared to stabilize during sustained agalsidase beta treatment. eGFR decline remained within normal ranges. This suggests that treatment may prevent further Fabry-related progression of cardiomyopathy in female patients and maintain normal kidney function.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/ehf2.12647","type":"journal-article","created":{"date-parts":[[2020,2,26]],"date-time":"2020-02-26T03:24:51Z","timestamp":1582687491000},"page":"825-834","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":26,"title":["Cardiomyopathy and Kidney Function in Agalsidase Beta-Treated Female Fabry Patients: A Pre-Treatment vs. Post-Treatment Analysis"],"prefix":"10.1093","volume":"7","author":[{"given":"Christoph","family":"Wanner","sequence":"first","affiliation":[{"name":"Department of Medicine, Division of Nephrology, University Hospital W\u00fcrzburg , W\u00fcrzburg 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Paris-Saclay University , Montigny ,","place":["France"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Derralynn A.","family":"Hughes","sequence":"additional","affiliation":[{"name":"Lysosomal Storage Disorder Unit, Royal Free London NHS Foundation Trust and, University College London , London ,","place":["UK"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John L.","family":"Jefferies","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Cardiovascular Diseases, University of Tennessee Health Science Center , Memphis, TN ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana Maria","family":"Martins","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, Federal University of S\u00e3o Paulo , S\u00e3o Paulo ,","place":["Brazil"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Albina","family":"Nowak","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, University Hospital of Z\u00fcrich, University of Z\u00fcrich , Z\u00fcrich ,","place":["Switzerland"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bojan","family":"Vujkovac","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, General Hospital Slovenj Gradec , Slovenj Gradec ,","place":["Slovenia"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frank","family":"Weidemann","sequence":"additional","affiliation":[{"name":"Medical Clinic I, Klinikum Vest, Knappschaftskrankenhaus , Recklinghausen ,","place":["Germany"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael L.","family":"West","sequence":"additional","affiliation":[{"name":"Department of Medicine, Division of Nephrology, Dalhousie University , Halifax, 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