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dc.contributorVall d'Hebron Barcelona Hospital Campus
dc.contributor.authorRamaswami, Uma
dc.contributor.authorBeck, Michael
dc.contributor.authorHughes, Derralynn
dc.contributor.authorKampmann, Christoph
dc.contributor.authorBotha, Jaco
dc.contributor.authorPintos Morell, Guillem
dc.date.accessioned2021-04-16T14:31:11Z
dc.date.available2021-04-16T14:31:11Z
dc.date.issued2019-10-25
dc.identifier.citationRamaswami U, Beck M, Hughes D, Kampmann C, Botha J, Pintos-Morell G, et al. Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: A 10-year fabry outcome survey (FOS) analysis. Drug Des Devel Ther. 2019;13:3705–15.
dc.identifier.issn1177-8881
dc.identifier.urihttp://hdl.handle.net/11351/5871
dc.descriptionAgalsidase alfa; Enzyme replacement therapy; Fabry disease
dc.description.abstractPurpose: Following the publication of 5-year agalsidase alfa enzyme replacement therapy (ERT) outcomes data from the Fabry Outcome Survey (FOS), 10-year data were analyzed. Patients and methods: FOS (ClinicalTrials.gov identifier: NCT03289065) data (April 2001 to August 2018) were retrospectively analyzed. Estimated glomerular filtration rate (eGFR) and left ventricular mass indexed to height (LVMI) were analyzed after treatment start (baseline) for patients with ≥3 measurements, including baseline and year 10. Results: Median (range) age (years) of the evaluable treated renal cohort at treatment start was 48.8 (17.9–67.3) for females (n=62), 34.4 (18.0–66.8) for males (n=90). With eGFR ≥60 mL/min/1.73 m2 at baseline, mean (95% CI) rate of eGFR change (eGFR/year) over 10 years was relatively stable in females (n=52; −0.55 [−1.12, +0.01]) and slightly declined in males (n=79; −1.99 [−2.45, −1.54]). With impaired kidney function (eGFR <60 mL/min/1.73 m2) at baseline, mean (95% CI) eGFR/year was stable in females (n=10; −0.14 [−1.43, +1.15]) and slightly declined in males (n=11; −2.79 [−4.01, −1.56]) over 10 years. Median (range) age (years) of the evaluable treated cardiac cohort at treatment start was 46.7 (3.7–67.3) for females (n=34), 28.2 (4.0–54.2) for males (n=35). With left ventricular hypertrophy (LVH; LVMI >48 g/m2.7 in females, >50 g/m2.7 in males) at baseline, mean (95% CI) LVMI/year slightly increased over 10 years in females (n=18; +1.51 [+0.91, +2.12]) and males (n=14; +0.87 (+0.19, +1.55). Without LVH at baseline, mean (95% CI) LVMI/year was stable in females (n=16; +0.52 [−0.13, +1.17]) and males (n=21; +0.57 [+0.02, +1.13]) over 10 years. Conclusion: Agalsidase alfa-treated patients with 10-year FOS data and preserved kidney function and/or normal LVMI at baseline remained largely stable; those with decreased kidney function or LVH at baseline experienced modest declines in renal function and/or increases in LVMI.
dc.language.isoeng
dc.publisherDove Medical Press
dc.relation.ispartofseriesDrug Design, Development and Therapy;13
dc.rightsAttribution-NonCommercial 4.0 International
dc.rights.urihttp://creativecommons.org/licenses/by-nc/4.0/
dc.sourceScientia
dc.subjectEnzims - Regulació
dc.subjectMalalties congènites
dc.subjectMetabolisme - Trastorns
dc.subject.meshFabry Disease
dc.subject.meshEnzyme Replacement Therapy
dc.titleCardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis
dc.typeinfo:eu-repo/semantics/article
dc.identifier.doi10.2147/DDDT.S207856
dc.subject.decsenfermedad de Fabry
dc.subject.decstratamiento de sustitución enzimática
dc.relation.publishversionhttps://www.dovepress.com/cardio--renal-outcomes-with-long--term-agalsidase-alfa-enzyme-replacem-peer-reviewed-article-DDDT
dc.type.versioninfo:eu-repo/semantics/publishedVersion
dc.audienceProfessionals
dc.contributor.organismesInstitut Català de la Salut
dc.contributor.authoraffiliation[Ramaswami U, Hughes D] Royal Free London NHS Foundation Trust, Lysosomal Disorders Unit, Institute of Immunity and Transplantation, London, UK. [Beck M, Kampmann C] Centre for Paediatric and Adolescent Medicine, University Medical Centre, University of Mainz, Mainz, Germany. [Botha J] Department of Biostatistics and Programming, Takeda, Zug, Switzerland. [Pintos-Morell G] Malalties minoritàries, Vall d’Hebron Hospital Universitari, Barcelona, Spain. Research Institute and Teaching Unit Germans Trias i Pujol. Universitat Autònoma de Barcelona, Bellaterra, Spain
dc.identifier.pmid31749608
dc.identifier.wos000492854800001
dc.rights.accessrightsinfo:eu-repo/semantics/openAccess


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