RT Journal Article SR Electronic T1 Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study JF Journal of Medical Genetics JO J Med Genet FD BMJ Publishing Group Ltd SP jmedgenet-2017-104863 DO 10.1136/jmedgenet-2017-104863 A1 Maarten Arends A1 Marieke Biegstraaten A1 Christoph Wanner A1 Sandra Sirrs A1 Atul Mehta A1 Perry M Elliott A1 Daniel Oder A1 Oliver T Watkinson A1 Daniel G Bichet A1 Aneal Khan A1 Mark Iwanochko A1 Frédéric M Vaz A1 André B P van Kuilenburg A1 Michael L West A1 Derralynn A Hughes A1 Carla E M Hollak YR 2018 UL http://jmg.bmj.com/content/early/2018/02/06/jmedgenet-2017-104863.abstract AB Background Two 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.Methods In 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.Results 387 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, P<0.001), persisting in the presence of antibodies. The risk to develop antibodies was higher for patients treated with agalsidase beta (OR 2.8, P=0.04). LVMI decreased in a higher proportion following the first year of agalsidase beta treatment (OR 2.27, P=0.03), while eGFR slopes were similar.Conclusions Treatment 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.