TY - JOUR 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 SP - 351 LP - 358 DO - 10.1136/jmedgenet-2017-104863 VL - 55 IS - 5 AU - Maarten Arends AU - Marieke Biegstraaten AU - Christoph Wanner AU - Sandra Sirrs AU - Atul Mehta AU - Perry M Elliott AU - Daniel Oder AU - Oliver T Watkinson AU - Daniel G Bichet AU - Aneal Khan AU - Mark Iwanochko AU - Frédéric M Vaz AU - André B P van Kuilenburg AU - Michael L West AU - Derralynn A Hughes AU - Carla E M Hollak Y1 - 2018/05/01 UR - http://jmg.bmj.com/content/55/5/351.abstract N2 - 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. ER -