Clinical outcomes in Fabry patients switching to agalsidase beta for renal ineffectiveness of the primary Fabry therapy: a single-centre analysis.

ABSTRACT:

Background

A treatment for classic Fabry disease (FD) should be defined effective on renal function when it maintains a decline of estimated glomerular filtration rate (eGFR) <1 ml/min/1.73 m²/year, and not effective when the eGFR loss remains ≥3 ml/min/1.73 m^2/year. Therefore, considering the evidence of dose-dependent efficacy of the enzyme replacement therapy (ERT) and the data reporting the disease progression after switching form agalsidase beta to migalastat, a switch to higher doses of ERT should be considered in adult Fabry patients who have an eGFR of 45-90 ml/min/1.73 m², with an eGFR slope ≥3 ml/min/1.73 m²/year. This study aimed to assess the effects of switching to agalsidase beta for renal ineffectiveness of the primary Fabr

SUBMITTER: Riccio E 

PROVIDER: S-EPMC12686814 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature