Time to treatment benefit for adult patients with Fabry disease receiving agalsidase beta: Data from the Fabry Registry

Result code in IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11110%2F16%3A10328562" target="_blank" >RIV/00216208:11110/16:10328562 - isvavai.cz</a>

Result on the web

<a href="http://dx.doi.org/10.1136/jmedgenet-2015-103486" target="_blank" >http://dx.doi.org/10.1136/jmedgenet-2015-103486</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1136/jmedgenet-2015-103486" target="_blank" >10.1136/jmedgenet-2015-103486</a>

Result language

angličtina

Original language name

Time to treatment benefit for adult patients with Fabry disease receiving agalsidase beta: Data from the Fabry Registry

Original language description

Background. Agalsidase β is a form of enzyme replacement therapy for Fabry disease, a genetic disorder characterised by low α-galactosidase A activity, accumulation of glycosphingolipids and life-threatening cardiovascular, renal and cerebrovascular events. In clinical trials, agalsidase β cleared glycolipid deposits from endothelial cells within 6 months; clearance from other cell types required sustained treatment. We hypothesised that there might be a 'lag time' to clinical benefit after initiating agalsidase β treatment, and analysed the incidence of severe clinical events over time in patients receiving agalsidase β. Methods: The incidence of severe clinical events (renal failure, cardiac events, stroke, death) was studied in 1044 adult patients (641 men, 403 women) enrolled in the Fabry Registry who received agalsidase β (average dose 1 mg/kg every 2 weeks) for up to 5 years. Results: The incidence of all severe clinical events was 111 per 1000 person-years (95% CI 84 to 145) during the first 6 months. After 6 months, the incidence decreased and remained stable within the range of 40-58 events per 1000 patient-years. The largest decrease in incidence rates was among male patients and those aged >=40 years when agalsidase β was initiated. Conclusions: Contrary to the expected increased incidence of severe clinical events with time, adult patients with Fabry disease had decreased incidence of severe clinical events after 6 months treatment with agalsidase β 1 mg/kg every 2 weeks.

Czech name

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Czech description

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Type

J_x - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)

CEP classification

FA - Cardiovascular diseases including cardio-surgery

OECD FORD branch

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Project

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Continuities

V - Vyzkumna aktivita podporovana z jinych verejnych zdroju

Publication year

2016

Confidentiality

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Name of the periodical

Journal of Medical Genetics

ISSN

0022-2593

e-ISSN

—

Volume of the periodical

53

Issue of the periodical within the volume

7

Country of publishing house

GB - UNITED KINGDOM

Number of pages

8

Pages from-to

495-502

UT code for WoS article

000378534500013

EID of the result in the Scopus database

2-s2.0-84979211037