Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064165%3A_____%2F22%3A10446851" target="_blank" >RIV/00064165:_____/22:10446851 - isvavai.cz</a>
Alternative codes found
RIV/00216208:11110/22:10446851
Result on the web
<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=qugYT4bM9" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=qugYT4bM9</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.3390/jcm11164810" target="_blank" >10.3390/jcm11164810</a>
Alternative languages
Result language
angličtina
Original language name
Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease
Original language description
Fabry disease is a rare lysosomal storage disorder caused by mutations in the GLA gene, which, without treatment, can cause significant renal dysfunction. We evaluated the effects of enzyme replacement therapy with agalsidase alfa on renal decline in patients with Fabry disease using data from the Fabry Outcome Survey (FOS) registry. Male patients with Fabry disease aged >16 years at agalsidase alfa start were stratified by low (<= 0.5 g/24 h) or high (>0.5 g/24 h) baseline proteinuria and by 'classic' or 'non-classic' phenotype. Overall, 193 male patients with low (n = 135) or high (n = 58) baseline proteinuria were evaluated. Compared with patients with low baseline proteinuria, those with high baseline proteinuria had a lower mean +/- standard deviation baseline eGFR (89.1 +/- 26.2 vs. 106.6 +/- 21.8 mL/min/1.73 m(2)) and faster mean +/- standard error eGFR decline (-3.62 +/- 0.42 vs. -1.61 +/- 0.28 mL/min/1.73 m(2) per year; p < 0.0001). Patients with classic Fabry disease had similar rates of eGFR decline irrespective of baseline proteinuria; only one patient with non-classic Fabry disease had high baseline proteinuria, preventing meaningful comparisons between groups. In this analysis, baseline proteinuria significantly impacted the rate of eGFR decline in the overall population, suggesting that early treatment with good proteinuria control may be associated with renoprotective effects.
Czech name
—
Czech description
—
Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
—
OECD FORD branch
30201 - Cardiac and Cardiovascular systems
Result continuities
Project
—
Continuities
V - Vyzkumna aktivita podporovana z jinych verejnych zdroju
Others
Publication year
2022
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Journal of Clinical Medicine [online]
ISSN
2077-0383
e-ISSN
2077-0383
Volume of the periodical
11
Issue of the periodical within the volume
16
Country of publishing house
CH - SWITZERLAND
Number of pages
15
Pages from-to
4810
UT code for WoS article
000845506800001
EID of the result in the Scopus database
2-s2.0-85138049221