Dose-Dependent Effect of Enzyme Replacement Therapy on Neutralizing Antidrug Antibody Titers and Clinical Outcome in Patients with Fabry Disease.

ABSTRACT:

Background

Use of enzyme replacement therapy (ERT) to treat Fabry disease, caused by deficient lysosomal α-galactosidase A activity, can lead to formation of neutralizing antidrug antibodies (ADAs). These antibodies are associated with increased accumulation of plasma globotriaosylceramide (Gb3) and disease progression. Because agalsidase ERT can saturate ADA-binding sites during infusions (achieving agalsidase/antibody equilibrium), we investigated in this open cohort study whether saturated patients (who have excess agalsidase after infusions) experience better clinical outcomes compared with not saturated patients (who have excess ADAs after infusions).

Methods

We isolated ADAs from sera of 26 men with Fabry disease receiving ERT (for a median of 94 months) and det

SUBMITTER: Lenders M 

PROVIDER: S-EPMC6287863 | biostudies-literature | 2018 Dec

REPOSITORIES: biostudies-literature