Renal Outcomes After Switching to Agalsidase Beta: A Single-Center Analysis

Renal Outcomes After Switching to Agalsidase Beta
in Fabry Disease: A Single-Center Analysis

• Renal involvement is a key feature of FD, characterized by Gb3 deposition in renal cells and progressive nephropathy that can lead to kidney failure by the third to fifth decade in untreated patients with classical mutations (1).

• Specific therapies have altered FD progression where ERT remains central, with agalsidase alfa, beta, and pegunigalsidase alfa (1).

• Since recent studies highlighted a dose-dependent effect of agalsidase, showing that higher doses enhance intracellular enzymatic activity, improving Gb3 clearance and slowing nephropathy progression (1).

• It has been recently suggested that a treatment for classic FD should be considered effective in preserving renal function when it maintains an eGFR decline <1 mL/min/1.73 m² per year (1).

• Included subjects were adult patients (≥18 years of age) with genetically determined FD, of both  genders, with eGFR between 45 and 90 mL/min/1.73 m², switched from at least 1 year of stable  treatment with the regular dose of agalsidase alfa or migalastat as their primary therapy, to agalsidase beta without any interval (1).

Study objective (1)

Study design and population (1)

• This single-centre observational study enrolled 11 adult patients (≥18 years) with genetically confirmed FD and baseline eGFR 45–90 mL/min/1.73 m².

• Patients meeting the inclusion criteria were adults of both genders who had received stable primary therapy for ≥1 year with agalsidase alfa or migalastat.

• The cohort was predominantly male (82%), with a mean age of 48.1 ± 11.0 years, and had received primary therapy for a mean duration of 55.3 ± 31.2 months; classical GLA variants were present in 82% and late-onset variants in 18%.

• Patients meeting inclusion criteria were followed for 24 months after the switch.

Study endpoints (1)

Study result (1)