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

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...

Authors: Lenders, Malte
Schmitz, Boris
Brand, Stefan-Martin
Brand, Eva
Division/Institute:FB 13: Biologie
FB 05: Medizinische Fakultät
Document types:Article
Media types:Text
Publication date:2018
Date of publication on miami:25.07.2019
Modification date:25.07.2019
Edition statement:[Electronic ed.]
Source:Orphanet Journal of Rare Diseases 13 (2018 ) 171, 1-2
Subjects:Enzyme replacement therapy; Longitudinal; Prospective
DDC Subject:610: Medizin und Gesundheit
License:CC BY 4.0
Language:English
Funding:Finanziert durch den Open-Access-Publikationsfonds 2018 der Deutschen Forschungsgemeinschaft (DFG) und der Westfälischen Wilhelms-Universität Münster (WWU Münster).
Format:PDF document
URN:urn:nbn:de:hbz:6-84129538247
Permalink:http://nbn-resolving.de/urn:nbn:de:hbz:6-84129538247
Other Identifiers:DOI: 10.1186/s13023-018-0916-1
Digital documents:artikel_lenders_2018.pdf

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.