{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,28]],"date-time":"2026-10-28T16:33:47Z","timestamp":1793205227760,"version":"4.5.0"},"reference-count":37,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"1","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2012,1]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background and objectives<\/jats:title>\n                    <jats:p>Fabry disease is a rare X-linked disease with multisystemic manifestations. This study investigated the effectiveness of long-term enzyme replacement therapy with agalsidase alfa in Fabry nephropathy treatment.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design, setting, participants, &amp; measurements<\/jats:title>\n                    <jats:p>\n                      In this observational study, data on patients receiving agalsidase alfa (0.2 mg\/kg every other week) were extracted from the Fabry Outcome Survey, an international registry of patients with Fabry disease. Serum creatinine and estimated GFR (eGFR) at baseline and after \u22655 years of treatment were assessed; 24-hour urinary protein excretion and BP measurements were also reviewed. The eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration formula. Patients with an eGFR &lt;30 ml\/min per 1.73 m\n                      <jats:sup>2<\/jats:sup>\n                      were excluded.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Renal function was assessed in 208 patients (mean enzyme replacement therapy, 7.4 years; range, 5.0\u201311.2 years). Mean yearly change in eGFR was \u22122.2 ml\/min per 1.73 m\n                      <jats:sup>2<\/jats:sup>\n                      in men and \u22120.7 ml\/min per 1.73 m\n                      <jats:sup>2<\/jats:sup>\n                      in women (95% confidence limits, \u22122.8; \u22121.7 and \u22121.4; 0.0, respectively). Patients with 24-hour protein excretion &gt;1 g\/24 h had poorer renal function at baseline and follow-up compared with patients with protein excretion of 500\u20131000 mg\/24 h or with proteinuria &lt;500 mg\/24 h. Renal function was worse in patients with baseline arterial hypertension, and there was a more rapid yearly decline compared with normotensive patients.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>This study suggests that long-term agalsidase alfa therapy is able to stabilize the rate of Fabry nephropathy progression in women and is associated with a mild to moderate decline of renal function in men.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.2215\/cjn.03130411","type":"journal-article","created":{"date-parts":[[2012,1,13]],"date-time":"2012-01-13T13:42:13Z","timestamp":1326462133000},"page":"60-69","source":"Crossref","is-referenced-by-count":68,"title":["The Effectiveness of Long-Term Agalsidase Alfa Therapy in the Treatment of Fabry Nephropathy"],"prefix":"10.2215","volume":"7","author":[{"given":"Sandro","family":"Feriozzi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joan","family":"Torras","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Markus","family":"Cybulla","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathy","family":"Nicholls","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gere","family":"Sunder-Plassmann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"West","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"276","reference":[{"key":"R1-12-20230811","doi-asserted-by":"crossref","first-page":"1600","DOI":"10.1093\/ndt\/gfm848","article-title":"Nephropathy in males and females with Fabry disease: Cross-sectional description of patients before treatment with enzyme replacement therapy.","volume":"23","author":"Ortiz","year":"2008","journal-title":"Nephrol Dial Transplant"},{"key":"R2-12-20230811","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1136\/hrt.2006.104026","article-title":"Effects of enzyme replacement therapy on the cardiomyopathy of Anderson-Fabry disease: A randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa.","volume":"94","author":"Hughes","year":"2008","journal-title":"Heart"},{"key":"R3-12-20230811","doi-asserted-by":"crossref","first-page":"1103","DOI":"10.1136\/hrt.2008.162800","article-title":"Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: A prospective long-term cardiac magnetic resonance imaging 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