Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease
Identifikátory výsledku
Kód výsledku v 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>
Nalezeny alternativní kódy
RIV/00216208:11110/22:10446851
Výsledek na webu
<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>
Alternativní jazyky
Jazyk výsledku
angličtina
Název v původním jazyce
Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease
Popis výsledku v původním jazyce
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.
Název v anglickém jazyce
Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease
Popis výsledku anglicky
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.
Klasifikace
Druh
J<sub>imp</sub> - Článek v periodiku v databázi Web of Science
CEP obor
—
OECD FORD obor
30201 - Cardiac and Cardiovascular systems
Návaznosti výsledku
Projekt
—
Návaznosti
V - Vyzkumna aktivita podporovana z jinych verejnych zdroju
Ostatní
Rok uplatnění
2022
Kód důvěrnosti údajů
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Údaje specifické pro druh výsledku
Název periodika
Journal of Clinical Medicine [online]
ISSN
2077-0383
e-ISSN
2077-0383
Svazek periodika
11
Číslo periodika v rámci svazku
16
Stát vydavatele periodika
CH - Švýcarská konfederace
Počet stran výsledku
15
Strana od-do
4810
Kód UT WoS článku
000845506800001
EID výsledku v databázi Scopus
2-s2.0-85138049221