{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,3]],"date-time":"2026-06-03T00:44:41Z","timestamp":1780447481254,"version":"3.54.1"},"reference-count":38,"publisher":"Oxford University Press (OUP)","issue":"11","license":[{"start":{"date-parts":[[2025,10,15]],"date-time":"2025-10-15T00:00:00Z","timestamp":1760486400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,11,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>A treatment for classic Fabry disease (FD) should be defined effective on renal function when it maintains a decline of estimated glomerular filtration rate (eGFR) &amp;lt;1 ml\/min\/1.73 m2\/year, and not effective when the eGFR loss remains \u22653 ml\/min\/1.73 m2\/year. Therefore, considering the evidence of dose-dependent efficacy of the enzyme replacement therapy (ERT) and the data reporting the disease progression after switching form agalsidase beta to migalastat, a switch to higher doses of ERT should be considered in adult Fabry patients who have an eGFR of 45\u201390 ml\/min\/1.73 m2, with an eGFR slope \u22653 ml\/min\/1.73 m2\/year. This study aimed to assess the effects of switching to agalsidase beta for renal ineffectiveness of the primary Fabry therapy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Data retrospectively taken during the pre-switch period were compared with data prospectively collected during the post-switch period. The primary endpoint was the effect on eGFR slope. Secondary endpoints were: changes in clinical events, 24-hour proteinuria, cardiac and neurologic parameters, FD-related symptoms, lyso-Gb3 plasma concentrations, and adverse events.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>In total, 11 patients (nine males and two females) switched to agalsidase beta from 55.3\u00a0\u00b1\u00a031.2 months of primary therapy with agalsidase alfa (eight patients) or migalastat (three patients), were evaluated for a follow-up period of 24 months. After the switch, a significant reduction of the eGFR slope was observed (\u22124.61 vs \u22120.45 ml\/min\/1.73 m2\/year, respectively in pre- and post-switch period; Ppost\/pre\u00a0&amp;lt;\u00a0.005). After the switch, plasma lyso-Gb3 levels progressively reduced, and the reduction reached the significance vs baseline at T2 (P\u00a0&amp;lt;\u00a0.05). Most of FD-related symptoms ameliorated during the primary Fabry therapy and remained stable after the switch. All other parameters were stable over time.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Treatment switch from agalsidase alfa or migalastat to agalsidase beta can attenuate eGFR decline and enhance lyso-Gb3 reduction, confirming the dose-dependent effect of agalsidase beta to further slow down FD progression.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1093\/ckj\/sfaf318","type":"journal-article","created":{"date-parts":[[2025,10,14]],"date-time":"2025-10-14T11:57:03Z","timestamp":1760443023000},"source":"Crossref","is-referenced-by-count":1,"title":["Clinical outcomes in Fabry patients switching to agalsidase beta for renal ineffectiveness of the primary Fabry therapy: a single-centre analysis"],"prefix":"10.1093","volume":"18","author":[{"given":"Eleonora","family":"Riccio","sequence":"first","affiliation":[{"name":"University Federico II of Naples Department of Public Health, Chair of Nephrology, , Naples ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivana","family":"Capuano","sequence":"additional","affiliation":[{"name":"University Federico II of Naples Department of Public Health, Chair of Nephrology, , Naples ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7933-6209","authenticated-orcid":false,"given":"Pasquale","family":"Buonanno","sequence":"additional","affiliation":[{"name":"University of Naples Federico II Department of Neurosciences, Reproductive and Odontostomatological Sciences, , Naples ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guido","family":"Iaccarino","sequence":"additional","affiliation":[{"name":"Federico II University Department of Clinical Medicine and Surgery, , Naples ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Calogero Lino","family":"Cirami","sequence":"additional","affiliation":[{"name":"Nephrology Dialysis Transplant Unit, Careggi Hospital , Florence ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renzo","family":"Mignani","sequence":"additional","affiliation":[{"name":"Nephrology, Dialysis and Transplantation, IRCCS S. Orsola University Hospital, University of Bologna , Bologna ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federico","family":"Pieruzzi","sequence":"additional","affiliation":[{"name":"Nephrology, Fondazione IRCCS San Gerardo dei Tintori , Monza ,","place":["Italy"]},{"name":"School of Medicine and Surgery, University of Milano-Bicocca , Milan ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Pisani","sequence":"additional","affiliation":[{"name":"University Federico II of Naples Department of Public Health, Chair of Nephrology, , Naples ,","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2025,10,15]]},"reference":[{"key":"2025111003420318900_bib1","doi-asserted-by":"publisher","first-page":"301","DOI":"10.1111\/cge.12386","article-title":"The kidney in Fabry disease","volume":"86","author":"Pisani","year":"2014","journal-title":"Clin Genet"},{"key":"2025111003420318900_bib2","doi-asserted-by":"publisher","first-page":"S139","DOI":"10.1097\/01.ASN.0000016683.73778.78","article-title":"Natural history and treatment of renal involvement in Fabry disease","volume":"13","author":"Branton","year":"2002","journal-title":"J Am Soc Nephrol"},{"key":"2025111003420318900_bib3","doi-asserted-by":"publisher","first-page":"274","DOI":"10.1159\/000502907","article-title":"Early biomarkers of Fabry nephropathy: a review of the literature","volume":"143","author":"Riccio","year":"2019","journal-title":"Nephron"},{"key":"2025111003420318900_bib4","doi-asserted-by":"publisher","first-page":"318","DOI":"10.1093\/ndt\/gfx009","article-title":"Parapelvic cysts, a distinguishing feature of renal Fabry disease","volume":"33","author":"Pisani","year":"2018","journal-title":"Nephrol Dial Transplant"},{"key":"2025111003420318900_bib5","doi-asserted-by":"publisher","first-page":"267","DOI":"10.1016\/j.ymgme.2012.08.003","article-title":"Enzyme replacement therapy in patients with Fabry disease: state of the art and review of the literature","volume":"107","author":"Pisani","year":"2012","journal-title":"Mol Genet Metab"},{"key":"2025111003420318900_bib6","doi-asserted-by":"publisher","first-page":"2743","DOI":"10.1001\/jama.285.21.2743","article-title":"Enzyme replacement therapy in Fabry disease: a randomized controlled trial","volume":"285","author":"Schiffmann","year":"2001","journal-title":"JAMA"},{"key":"2025111003420318900_bib7","doi-asserted-by":"publisher","first-page":"9","DOI":"10.1056\/NEJM200107053450102","article-title":"International collaborative Fabry disease study group: safety and efficacy of recombinant human alpha-galactosidase a replacement therapy in Fabry\u2019s disease","volume":"345","author":"Eng","year":"2001","journal-title":"N Engl J Med"},{"key":"2025111003420318900_bib8","doi-asserted-by":"publisher","first-page":"653","DOI":"10.1681\/ASN.2013121322","article-title":"Fabry disease: dose matters","volume":"25","author":"Warnock","year":"2014","journal-title":"J Am Soc Nephrol"},{"key":"2025111003420318900_bib9","doi-asserted-by":"publisher","first-page":"2879","DOI":"10.1681\/ASN.2018070740","article-title":"Dose dependent effect of Enzyme replacement therapy on neutralizing antidrug antibody titers and clinical outcome in patients with Fabry disease","volume":"29","author":"Lenders","year":"2018","journal-title":"J Am Soc Nephrol"},{"key":"2025111003420318900_bib10","doi-asserted-by":"publisher","first-page":"1576","DOI":"10.1681\/ASN.2006111263","article-title":"Weekly enzyme replacement therapy may slow decline of renal function in patients with Fabry disease who are on long-term biweekly dosing","volume":"18","author":"Schiffmann","year":"2007","journal-title":"J Am Soc Nephrol"},{"key":"2025111003420318900_bib11","doi-asserted-by":"publisher","first-page":"1362","DOI":"10.1093\/ndt\/gfx319","article-title":"Fabry disease under enzyme replacement therapy-new insights in efficacy of different dosages","volume":"33","author":"Kr\u00e4mer","year":"2018","journal-title":"Nephrol Dial Transplant"},{"key":"2025111003420318900_bib12","doi-asserted-by":"publisher","first-page":"952","DOI":"10.1681\/ASN.2015030337","article-title":"Patients with Fabry disease after enzyme replacement therapy dose reduction and switch-2-year follow-up","volume":"27","author":"Lenders","year":"2016","journal-title":"J Am Soc Nephrol"},{"key":"2025111003420318900_bib13","doi-asserted-by":"publisher","first-page":"371","DOI":"10.1111\/cge.14266","article-title":"New insights in efficacy of different enzyme replacement therapy dosages in Fabry disease: switch studies data following agalsidase beta shortage","volume":"103","author":"Riccio","year":"2023","journal-title":"Clin Genet"},{"key":"2025111003420318900_bib14","doi-asserted-by":"publisher","first-page":"545","DOI":"10.1056\/NEJMoa1510198","article-title":"Treatment of Fabry\u2019s disease with the pharmacologic chaperone migalastat","volume":"375","author":"Germain","year":"2016","journal-title":"N Engl J Med"},{"key":"2025111003420318900_bib15","doi-asserted-by":"publisher","first-page":"288","DOI":"10.1136\/jmedgenet-2016-104178","article-title":"Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study","volume":"54","author":"Hughes","year":"2017","journal-title":"J Med Genet"},{"key":"2025111003420318900_bib16","doi-asserted-by":"publisher","first-page":"e12771","DOI":"10.1002\/jimd.12771","article-title":"Renal and multisystem effectiveness of 3.9 years of migalastat in a global real-world cohort: results from the FollowME Fabry pathfinders registry","volume":"48","author":"Hughes","year":"2025","journal-title":"J Inher Metab Disea"},{"key":"2025111003420318900_bib17","doi-asserted-by":"crossref","first-page":"1224","DOI":"10.1002\/cpt.1321","article-title":"Oral chaperone therapy migalastat for treating Fabry disease: enzymatic response and serum biomarker changes after 1 year","volume":"105","author":"M\u00fcntze","year":"2019","journal-title":"Clin Pharmacol Ther"},{"key":"2025111003420318900_bib18","doi-asserted-by":"publisher","first-page":"1080","DOI":"10.1002\/jimd.12773","article-title":"Clinical outcomes in patients switching from agalsidase beta to migalastat: a Fabry registry analysis","volume":"47","author":"Pisani","year":"2024","journal-title":"J Inher Metab Disea"},{"key":"2025111003420318900_bib19","doi-asserted-by":"publisher","first-page":"272","DOI":"10.1093\/ehjcvp\/pvab025","article-title":"Treatment of Fabry disease management with migalastat-outcome from a prospective 24 months observational multicenter study (FAMOUS)","volume":"8","author":"Lenders","year":"2022","journal-title":"Eur Heart J Cardiovasc Pharmacother"},{"key":"2025111003420318900_bib20","doi-asserted-by":"publisher","first-page":"e0173358","DOI":"10.1371\/journal.pone.0173358","article-title":"Enzyme replacement therapy for Anderson-Fabry disease: a complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies","volume":"12","author":"El Dib","year":"2017","journal-title":"PLoS ONE"},{"key":"2025111003420318900_bib21","doi-asserted-by":"publisher","first-page":"3705","DOI":"10.2147\/DDDT.S207856","article-title":"Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: a 10-year Fabry outcome survey (FOS) analysis","volume":"13","author":"Ramaswami","year":"2019","journal-title":"DDDT"},{"key":"2025111003420318900_bib22","doi-asserted-by":"publisher","first-page":"1136\u201346","DOI":"10.1093\/ckj\/sfaa065","article-title":"Agalsidase beta treatment slows estimated glomerular filtration rate loss in classic Fabry disease patients: results from an individual patient data meta-analysis","volume":"14","author":"Ortiz","year":"2021","journal-title":"Clin Kidney J"},{"key":"2025111003420318900_bib23","doi-asserted-by":"publisher","first-page":"1867","DOI":"10.1038\/s41591-023-02418-0","article-title":"A meta-analysis of GFR slope as a surrogate end-point for kidney failure","volume":"29","author":"Levey","year":"2023","journal-title":"Nat Med"},{"key":"2025111003420318900_bib24","doi-asserted-by":"publisher","first-page":"1735","DOI":"10.1681\/ASN.2019010007","article-title":"GFR slope as a surrogate end point for kidney disease progression in clinical trials: a meta-analysis of treatment effects of randomized controlled trials","volume":"30","author":"Inker","year":"2019","journal-title":"J Am Soc Nephrol"},{"key":"2025111003420318900_bib25","doi-asserted-by":"publisher","first-page":"1746","DOI":"10.1681\/ASN.2019010008","article-title":"Evaluating glomerular filtration rate slope as a surrogate end point for ESKD in clinical trials: an individual participant meta-analysis of observational data","volume":"30","author":"Grams","year":"2019","journal-title":"J Am Soc Nephrol"},{"key":"2025111003420318900_bib26","doi-asserted-by":"publisher","first-page":"1756","DOI":"10.1681\/ASN.2019010009","article-title":"Performance of GFR slope as a surrogate endpoint for kidney disease progression in clinical trials: a statistical simulation","volume":"30","author":"Greene","year":"2019","journal-title":"J Am Soc Nephrol"},{"key":"2025111003420318900_bib27","doi-asserted-by":"publisher","first-page":"18","DOI":"10.1093\/ndt\/gfad164","article-title":"Interpretation of GFR slope in untreated and treated adult Fabry patients","volume":"39","author":"Pisani","year":"2023","journal-title":"Nephrol Dial Transplant"},{"key":"2025111003420318900_bib28","doi-asserted-by":"publisher","first-page":"49","DOI":"10.1016\/j.ymgme.2022.07.010","article-title":"An expert consensus on practical clinical recommendations and guidance for patients with classic Fabry disease","volume":"137","author":"Germain","year":"2022","journal-title":"Mol Genet Metab"},{"key":"2025111003420318900_bib29","doi-asserted-by":"publisher","first-page":"10","DOI":"10.1159\/000493469","article-title":"Glomerular hyperfiltration: an early marker of nephropathy in Fabry disease","volume":"141","author":"Riccio","year":"2019","journal-title":"Nephron"},{"key":"2025111003420318900_bib30","doi-asserted-by":"publisher","first-page":"19","DOI":"10.1007\/s11547-011-0710-9","article-title":"Role of cardiac MRI in evaluating patients with Anderson-Fabry disease: assessing cardiac effects of long-term enzyme replacement therapy","volume":"117","author":"Messalli","year":"2012","journal-title":"Radiol Med"},{"key":"2025111003420318900_bib31","doi-asserted-by":"publisher","first-page":"563","DOI":"10.1007\/s00234-017-1829-8","article-title":"Corpus callosum involvement: a useful clue for differentiating Fabry disease from multiple sclerosis","volume":"59","author":"Cocozza","year":"2017","journal-title":"Neuroradiology"},{"key":"2025111003420318900_bib32","doi-asserted-by":"publisher","first-page":"1077\u201388","DOI":"10.1007\/s13244-018-0664-8","article-title":"Neuroimaging in Fabry disease: current knowledge and future directions","volume":"9","author":"Cocozza","year":"2018","journal-title":"Insights Imaging"},{"key":"2025111003420318900_bib33","first-page":"41","article-title":"Effects of switching from agalsidase beta to agalsidase alfa in 10 patients with Anderson-Fabry disease","volume":"9","author":"Pisani","year":"2013","journal-title":"JIMD Rep"},{"key":"2025111003420318900_bib34","doi-asserted-by":"publisher","first-page":"206","DOI":"10.3390\/ijms22010206","article-title":"Fabry disease therapy: state-of-the-art and current challenges","volume":"22","author":"Azevedo","year":"2020","journal-title":"Int J Mol Sci"},{"key":"2025111003420318900_bib35","author":"Agalsidase beta FDA full approval","year":"2025"},{"key":"2025111003420318900_bib36","doi-asserted-by":"publisher","first-page":"173","DOI":"10.1016\/j.ymgme.2022.08.003","article-title":"Algasidase-\u03b2 should be proposed as first line therapy in classic male Fabry patients with undetectable \u03b1-galactosidase a activity","volume":"137","author":"Nowak","year":"2022","journal-title":"Mol Genet Metab"},{"key":"2025111003420318900_bib37","doi-asserted-by":"crossref","first-page":"85895","DOI":"10.18632\/oncotarget.13135","article-title":"Genetic variants associated with Fabry disease progression despite enzyme replacement therapy","volume":"7","author":"Scionti","year":"2016","journal-title":"Oncotarget"},{"key":"2025111003420318900_bib38","doi-asserted-by":"publisher","first-page":"73","DOI":"10.2147\/PPA.S54922","article-title":"Efficacy of a reduced pill burden on therapeutic adherence to calcineurin inhibitors in renal transplant recipients: an observational study","volume":"8","author":"Sabbatini","year":"2014","journal-title":"Patient Prefer Adherence"}],"container-title":["Clinical Kidney Journal"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/academic.oup.com\/ckj\/advance-article-pdf\/doi\/10.1093\/ckj\/sfaf318\/64711474\/sfaf318.pdf","content-type":"application\/pdf","content-version":"am","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/ckj\/article-pdf\/18\/11\/sfaf318\/64711474\/sfaf318.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/academic.oup.com\/ckj\/article-pdf\/18\/11\/sfaf318\/64711474\/sfaf318.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,10]],"date-time":"2025-11-10T08:42:12Z","timestamp":1762764132000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/ckj\/article\/doi\/10.1093\/ckj\/sfaf318\/8286902"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,10,15]]},"references-count":38,"journal-issue":{"issue":"11","published-print":{"date-parts":[[2025,11,3]]}},"URL":"https:\/\/doi.org\/10.1093\/ckj\/sfaf318","relation":{},"ISSN":["2048-8505","2048-8513"],"issn-type":[{"value":"2048-8505","type":"print"},{"value":"2048-8513","type":"electronic"}],"subject":[],"published-other":{"date-parts":[[2025,11]]},"published":{"date-parts":[[2025,10,15]]},"article-number":"sfaf318"}}