Clinical kidney journal · 2025
Clinical outcomes in Fabry patients switching to agalsidase beta for renal ineffectiveness of the primary Fabry therapy: a single-centre analysis.
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Riccio Eleonora, Capuano Ivana, Buonanno Pasquale, Iaccarino Guido, Cirami Calogero Lino, Mignani Renzo, Pieruzzi Federico, Pisani Antonio
Аннотация
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 m2/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 Fabry therapy.
METHODS: Data retrospectively taken during the pre-switch period were compared with data prospectively collected during the post-switch period. The primary endpoint was the effect on eGFR slope. Secondary endpoints were: changes in clinical events, 24-hour proteinuria, cardiac and neurologic parameters, FD-related symptoms, lyso-Gb3 plasma concentrations, and adverse events.
RESULTS: In total, 11 patients (nine males and two females) switched to agalsidase beta from 55.3 ± 31.2 months of primary therapy with agalsidase alfa (eight patients) or migalastat (three patients), were evaluated for a follow-up period of 24 months. After the switch, a significant reduction of the eGFR slope was observed (-4.61 vs -0.45 ml/min/1.73 m2/year, respectively in pre- and post-switch period; P post /pre < .005). After the switch, plasma lyso-Gb3 levels progressively reduced, and the reduction reached the significance vs baseline at T2 (P < .05). Most of FD-related symptoms ameliorated during the primary Fabry therapy and remained stable after the switch. All other parameters were stable over time.
CONCLUSIONS: Treatment switch from agalsidase alfa or migalastat to agalsidase beta can attenuate eGFR decline and enhance lyso-Gb3 reduction, confirming the dose-dependent effect of agalsidase beta to further slow down FD progression.
METHODS: Data retrospectively taken during the pre-switch period were compared with data prospectively collected during the post-switch period. The primary endpoint was the effect on eGFR slope. Secondary endpoints were: changes in clinical events, 24-hour proteinuria, cardiac and neurologic parameters, FD-related symptoms, lyso-Gb3 plasma concentrations, and adverse events.
RESULTS: In total, 11 patients (nine males and two females) switched to agalsidase beta from 55.3 ± 31.2 months of primary therapy with agalsidase alfa (eight patients) or migalastat (three patients), were evaluated for a follow-up period of 24 months. After the switch, a significant reduction of the eGFR slope was observed (-4.61 vs -0.45 ml/min/1.73 m2/year, respectively in pre- and post-switch period; P post /pre < .005). After the switch, plasma lyso-Gb3 levels progressively reduced, and the reduction reached the significance vs baseline at T2 (P < .05). Most of FD-related symptoms ameliorated during the primary Fabry therapy and remained stable after the switch. All other parameters were stable over time.
CONCLUSIONS: Treatment switch from agalsidase alfa or migalastat to agalsidase beta can attenuate eGFR decline and enhance lyso-Gb3 reduction, confirming the dose-dependent effect of agalsidase beta to further slow down FD progression.