{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,7]],"date-time":"2026-11-07T02:54:13Z","timestamp":1794020053025,"version":"4.8.0"},"reference-count":42,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T00:00:00Z","timestamp":1762387200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T00:00:00Z","timestamp":1762387200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Eur J Clin Investigation"],"published-print":{"date-parts":[[2026,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>We present the final report from the Fabry Outcome Survey (FOS) on long\u2010term effectiveness and safety of agalsidase alfa in adults (\u226518\u2009years old).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>FOS was an international, multicentre, observational registry (NCT03289065), designed to enhance the understanding of Fabry disease and improve clinical management. Primary effectiveness endpoints were annualized change in estimated glomerular filtration rate (eGFR) and left ventricular mass index (LVMI), and time to and age at composite morbidity event (comprising renal, cardiac or stroke events) and death. Safety outcomes were also assessed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      FOS included data for 1864 adults (female\/male,\n                      <jats:italic>n\u2009=<\/jats:italic>\n                      \u2009907\/957) who received agalsidase alfa only for a median (minimum, maximum) of 6.0 (0, 21.6) years, and 1613 untreated adults (female\/male,\n                      <jats:italic>n\u2009=<\/jats:italic>\n                      \u20091235\/378). At baseline, mean (standard deviation [SD]) eGFR was 94.01 (27.60) mL\/min\/1.73\u2009m\n                      <jats:sup>2<\/jats:sup>\n                      in treated adults; annualized changes in eGFR (slope [standard error; SE]) remained relatively stable in females and declined slightly in males (\u22121.07 [.12] vs. \u22122.17 [.12] mL\/min\/1.73\u2009m\n                      <jats:sup>2<\/jats:sup>\n                      ). At baseline, mean (SD) LVMI was 58.25 (25.01) g\/m\n                      <jats:sup>2.7<\/jats:sup>\n                      and LVMI (slope [SE]) remained stable (.34 [.16] vs. .38 [.15] g\/m\n                      <jats:sup>2.7<\/jats:sup>\n                      \/year in females and males, respectively). Time (median [95% confidence interval]) from treatment initiation to first composite event was longer for females than males (83.4 [65.7\u201398.0] vs. 56.3 [45.6\u201366.7] months); age (median [minimum, maximum]) at death was also higher for treated females than males (69.9 [32.5, 87.7] vs. 59.1 [26.2, 79.6] years). Agalsidase alfa was generally well tolerated.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>This report further supports the long\u2010term effectiveness and safety of agalsidase alfa in adults with Fabry disease.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/eci.70142","type":"journal-article","created":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T14:46:18Z","timestamp":1762440378000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Long\u2010term effectiveness and safety outcomes in adults with Fabry disease treated with agalsidase alfa: 20\u2009years of data from the Fabry Outcome Survey"],"prefix":"10.1111","volume":"56","author":[{"given":"Derralynn A.","family":"Hughes","sequence":"first","affiliation":[{"name":"Royal Free London NHS Foundation Trust University College London  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guillem","family":"Pintos\u2010Morell","sequence":"additional","affiliation":[{"name":"Vall d'Hebron Research Institute  Barcelona Spain"},{"name":"Medical Consulting Committee, MPS\u2010Lisosomales Association  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christoph","family":"Kampmann","sequence":"additional","affiliation":[{"name":"Johannes Gutenberg School of Medicine University of Mainz  Mainz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina","family":"Anagnostopoulou","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals International AG  Zurich Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8974-2438","authenticated-orcid":false,"given":"Jaco","family":"Botha","sequence":"additional","affiliation":[{"name":"Takeda Pharmaceuticals International AG  Zurich Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Siddharth","family":"Jain","sequence":"additional","affiliation":[{"name":"Takeda Development Center Americas, Inc.  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