Deep characterization of the anti-drug antibodies developed in Fabry disease patients, a prospective analysis from the French multicenter cohort FFABRY

Affiliation auteurs!!!! Error affiliation !!!!
TitreDeep characterization of the anti-drug antibodies developed in Fabry disease patients, a prospective analysis from the French multicenter cohort FFABRY
Type de publicationJournal Article
Year of Publication2018
AuteursMauhin W, Lidove O, Amelin D, Lamari F, Caillaud C, Mingozzi F, Dzangue-Tchoupou G, Arouche-Delaperche L, Douillard C, Dussol B, Leguy-Seguin V, D'Halluin P, Noel E, Zenone T, Matignon M, Maillot F, Ly K-H, Besson G, Willems M, Labombarda F, Masseau A, Lavigne C, Froissart R, Lacombe D, Ziza JMarc, Hachulla E, Benveniste O
JournalORPHANET JOURNAL OF RARE DISEASES
Volume13
Pagination127
Date PublishedJUL 31
Type of ArticleArticle
ISSN1750-1172
Mots-clésAgalsidase, Anti-drug antibodies, enzyme replacement therapy, Fabry disease, IgG4, Lysosomal storage disease
Résumé

{Background: Fabry disease (OMIM \#301500) is an X-linked disorder caused by alpha-galactosidase A deficiency with two major clinical phenotypes: classic and non-classic of different prognosis. From 2001, enzyme replacement therapies (ERT) have been available. We aimed to determine the epidemiology and the functional characteristics of anti-drug antibodies. Patients from the French multicenter cohort FFABRY (n = 103 patients, 53 males) were prospectively screened for total anti-agalsidase IgG and IgG subclasses with a home-made enzyme-linked immunosorbent assay (ELISA), enzyme-inhibition assessed with neutralization assays and lysoGb3 plasma levels, and compared for clinical outcomes. Results: Among the patients exposed to agalsidase, 40% of men (n = 18/45) and 8% of women (n = 2/25) had antibodies with a complete cross-reactivity towards both ERTs. Antibodies developed preferentially in men with non-missense GM mutations (relative risk 2.88
DOI10.1186/s13023-018-0877-4