1. Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study
- Author
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Mark R. Iwanochko, Sandra Sirrs, Michael West, Perry M. Elliott, Frédéric M. Vaz, Marieke Biegstraaten, Maarten Arends, Christoph Wanner, Derralynn Hughes, Oliver Watkinson, Carla E. M. Hollak, André B.P. van Kuilenburg, Atul Mehta, Aneal Khan, Daniel G. Bichet, Daniel Oder, Graduate School, Other departments, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, Laboratory Genetic Metabolic Diseases, Endocrinology, APH - Personalized Medicine, and APH - Methodology
- Subjects
0301 basic medicine ,Adult ,Male ,Pediatrics ,medicine.medical_specialty ,Endocrinology, Diabetes and Metabolism ,Urology ,Renal function ,Therapeutics ,Biochemistry ,Cohort Studies ,03 medical and health sciences ,0302 clinical medicine ,Endocrinology ,Genetics ,medicine ,Humans ,Enzyme Replacement Therapy ,ert ,Beta (finance) ,Molecular Biology ,Genetics (clinical) ,Retrospective Studies ,fabry disease ,agalsidase alfa ,business.industry ,Retrospective cohort study ,Enzyme replacement therapy ,Middle Aged ,medicine.disease ,Fabry disease ,Recombinant Proteins ,AGALSIDASE BETA ,Isoenzymes ,030104 developmental biology ,Treatment Outcome ,alpha-Galactosidase ,Propensity score matching ,agalsidase beta ,Female ,business ,Agalsidase alfa ,030217 neurology & neurosurgery ,Cohort study ,Glomerular Filtration Rate - Abstract
BackgroundTwo recombinant enzymes (agalsidase alfa 0.2 mg/kg/every other week and agalsidase beta 1.0 mg/kg/every other week) have been registered for the treatment of Fabry disease (FD), at equal high costs. An independent international initiative compared clinical and biochemical outcomes of the two enzymes.MethodsIn this multicentre retrospective cohort study, clinical event rate, left ventricular mass index (LVMI), estimated glomerular filtration rate (eGFR), antibody formation and globotriaosylsphingosine (lysoGb3) levels were compared between patients with FD treated with agalsidase alfa and beta at their registered dose after correction for phenotype and sex.Results387 patients (192 women) were included, 248 patients received agalsidase alfa. Mean age at start of enzyme replacement therapy was 46 (±15) years. Propensity score matched analysis revealed a similar event rate for both enzymes (HR 0.96, P=0.87). The decrease in plasma lysoGb3 was more robust following treatment with agalsidase beta, specifically in men with classical FD (β: −18 nmol/L, PConclusionsTreatment with agalsidase beta at higher dose compared with agalsidase alfa does not result in a difference in clinical events, which occurred especially in those with more advanced disease. A greater biochemical response, also in the presence of antibodies, and better reduction in left ventricular mass was observed with agalsidase beta.
- Published
- 2018