Clinical outcomes in patients switching from agalsidase beta to migalastat: A Fabry Registry analysis

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064165%3A_____%2F24%3A10483176" target="_blank" >RIV/00064165:_____/24:10483176 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00216208:11110/24:10483176

Výsledek na webu

<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=CgD0jo3Ivt" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=CgD0jo3Ivt</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1002/jimd.12773" target="_blank" >10.1002/jimd.12773</a>

Jazyk výsledku

angličtina

Název v původním jazyce

Clinical outcomes in patients switching from agalsidase beta to migalastat: A Fabry Registry analysis

Popis výsledku v původním jazyce

Fabry Registry data were analyzed among 83 agalsidase beta-treated patients with Fabry disease who switched to migalastat. Outcomes (estimated glomerular filtration rate [eGFR], urine protein-creatinine ratio [UPCR], plasma globotriaosylceramide [GL-3], plasma globotriaosylsphingosine [lyso-GL-3], interventricular septal wall thickness [IVST], left posterior wall thickness [LPWT], left ventricular mass index [LVMI]) were assessed using linear mixed models to estimate annual change over time in the pre- and postswitch periods. eGFR decreased throughout both periods (preswitch: -0.85 mL/min/1.73 m(2)/year; postswitch: -1.96 mL/min/1.73 m(2)/year; both p &lt; 0.0001), with steeper decline postswitch (p(pre/post) = 0.01) in both classic and late-onset patients. UPCR increased significantly postswitch (p(pre/post) = 0.003) among classic patients and was stable in both periods among late-onset patients. GL-3 trajectories worsened postswitch across phenotypes (p(pre/post) = 0.0005 classic, 0.02 late-onset). LPWT was stable preswitch (0.07 mm/year, p = 0.25) and decreased postswitch (-0.51 mm/year, p = 0.0005; p(pre/post) = 0.0009), primarily among late-onset patients. IVST and LVMI slopes varied significantly by phenotype. Among classic patients, IVST and LVMI were stable and decreasing, respectively preswitch and increasing postswitch (p(pre/post) = 0.02 IVST, 0.01 LVMI). Among late-onset patients, IVST significantly decreased postswitch (p(pre/post) = 0.0003); LVMI was stable over time (p(pre/post) = 0.89). Ultimately, eGFR and GL-3 trajectories worsened postswitch across phenotypes, while UPCR and cardiac measures worsened among classic and stabilized/improved among late-onset patients. These findings indicate variability in long-term outcomes after switching from ERT to migalastat, underscoring the importance of careful monitoring.

Název v anglickém jazyce

Clinical outcomes in patients switching from agalsidase beta to migalastat: A Fabry Registry analysis

Popis výsledku anglicky

Fabry Registry data were analyzed among 83 agalsidase beta-treated patients with Fabry disease who switched to migalastat. Outcomes (estimated glomerular filtration rate [eGFR], urine protein-creatinine ratio [UPCR], plasma globotriaosylceramide [GL-3], plasma globotriaosylsphingosine [lyso-GL-3], interventricular septal wall thickness [IVST], left posterior wall thickness [LPWT], left ventricular mass index [LVMI]) were assessed using linear mixed models to estimate annual change over time in the pre- and postswitch periods. eGFR decreased throughout both periods (preswitch: -0.85 mL/min/1.73 m(2)/year; postswitch: -1.96 mL/min/1.73 m(2)/year; both p &lt; 0.0001), with steeper decline postswitch (p(pre/post) = 0.01) in both classic and late-onset patients. UPCR increased significantly postswitch (p(pre/post) = 0.003) among classic patients and was stable in both periods among late-onset patients. GL-3 trajectories worsened postswitch across phenotypes (p(pre/post) = 0.0005 classic, 0.02 late-onset). LPWT was stable preswitch (0.07 mm/year, p = 0.25) and decreased postswitch (-0.51 mm/year, p = 0.0005; p(pre/post) = 0.0009), primarily among late-onset patients. IVST and LVMI slopes varied significantly by phenotype. Among classic patients, IVST and LVMI were stable and decreasing, respectively preswitch and increasing postswitch (p(pre/post) = 0.02 IVST, 0.01 LVMI). Among late-onset patients, IVST significantly decreased postswitch (p(pre/post) = 0.0003); LVMI was stable over time (p(pre/post) = 0.89). Ultimately, eGFR and GL-3 trajectories worsened postswitch across phenotypes, while UPCR and cardiac measures worsened among classic and stabilized/improved among late-onset patients. These findings indicate variability in long-term outcomes after switching from ERT to migalastat, underscoring the importance of careful monitoring.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30201 - Cardiac and Cardiovascular systems

Projekt

—

Návaznosti

N - Vyzkumna aktivita podporovana z neverejnych zdroju

Rok uplatnění

2024

Kód důvěrnosti údajů

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

Název periodika

Journal of Inherited Metabolic Disease

ISSN

0141-8955

e-ISSN

1573-2665

Svazek periodika

47

Číslo periodika v rámci svazku

5

Stát vydavatele periodika

NL - Nizozemsko

Počet stran výsledku

16

Strana od-do

1080-1095

Kód UT WoS článku

001262378100001

EID výsledku v databázi Scopus

2-s2.0-85197377666