Fabry disease biomarkers in patients switched from enzyme-replacement therapy to migalastat oral chaperone therapy.
Bioanalysis
; 15(23): 1421-1437, 2023 Dec.
Article
em En
| MEDLINE
| ID: mdl-37847061
ABSTRACT
Background:
A biomarker profile was evaluated longitudinally in patients with Fabry disease switched from enzyme-replacement therapy (ERT) to migalastat.Methods:
16 Gb3 isoforms and eight lyso-Gb3 analogues were analyzed in plasma and urine by LC-MS/MS at baseline and at three different time points in naive participants and participants switching from either agalsidase α or ß to migalastat.Results:
29 adult participants were recruited internationally (seven centers). The Mainz Severity Score Index and mean biomarker levels remained stable (p ≥ 0.05) over a minimum of 12 months compared with baseline following the treatment switch.Conclusion:
In this cohort of patients with Fabry disease with amenable mutations, in the short term, a switch from ERT to migalastat did not have a marked effect on the average biomarker profile.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Doença de Fabry
Idioma:
En
Ano de publicação:
2023
Tipo de documento:
Article