Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study.

ABSTRACT:

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 s

SUBMITTER: Arends M 

PROVIDER: S-EPMC5931248 | biostudies-literature | 2018 May

REPOSITORIES: biostudies-literature