Neutralizing anti-drug antibodies in Fabry disease have no obvious clinical impact?

Malte LendersEmail author View ORCID ID profile,
Boris Schmitz,
Stefan-Martin Brand and
Eva Brand

Orphanet Journal of Rare Diseases201813:171

https://doi.org/10.1186/s13023-018-0916-1

Received: 27 August 2018
Accepted: 21 September 2018
Published: 29 September 2018

Abstract

Fabry disease (FD) is a rare X-linked disorder caused by a deficiency of lysosomal α-galactosidase A activity. Treatment with recombinant enzyme replacement therapy is available since 2001 and the effects of anti-drug antibodies (ADA) on therapy efficacy and disease outcome in affected patients have been controversially reported. In this letter we discuss the importance of adequate measurements of neutralizing ADAs and appropriate longitudinal analysis to determine therapy efficiency and clinical outcome in patients with FD.