Assessment and impact of dose escalation on anti-drug antibodies in Fabry disease.

ABSTRACT: Enzyme replacement therapy (ERT) with recombinant α-galactosidase A (AGAL) can lead to the formation of neutralizing anti-drug antibodies (ADA), which significantly limit treatment efficacy in patients with Fabry disease (FD). The effects of dose escalation on ADA titer and plasma globotriaosylsphingosine (lyso-Gb₃) level are unknown. We screened 250 FD patients (200 males, 50 females) under ERT for ADAs and assessed the impact of an approved dose escalation in affected patients, focusing on ADA titers and plasma lyso-Gb₃. ADA-positive patients were identified by serum-mediated inhibition assays, followed by titration assays to determine the individual inhibitory capacities of ADAs against agalsidase-alfa and agalsidase-beta. 70 (35%) of the male patients were ADA-pos

SUBMITTER: Lenders M 

PROVIDER: S-EPMC9782970 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature