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Safety and Tolerability of a Shorter Agalsidase Beta Infusion Time in Patients with Classic or Later-Onset Fabry
Dominique P Germain1,2,3, Alice Porto Vasconcelos3, Lien Tran Thi Phuong2
1Division of Medical Genetics, University of Versailles-St Quentin en Yvelines (UVSQ), Paris-Saclay University, 2 Avenue de la Source de la Bièvre, F-78180 Montigny, France.
Background:
The multisystem manifestations of Fabry disease can create major challenges in patient care. Although enzyme replacement therapy with recombinant agalsidase beta has demonstrated clinical benefits, the standard fortnightly, multi-hour infusion regimen imposes a substantial burden on patients.
Methods:
We assessed the safety and feasibility of shortening the agalsidase beta infusion time to 90 min in adult patients with classic or later-onset Fabry disease in the absence of premedication. A total of 39 consecutive adult patients (agalsidase-naïve: n = 7; with significant comorbidities: n = 15) with no recent infusion-associated reactions underwent a total of 85 agalsidase beta infusions in our tertiary reference centre for lysosomal diseases. Each infusion was administered at a constant rate (between 0.78 and 1.17 mg/min, depending on the total dose administered).
Results:
No adverse events of any type (including discomfort and infusion-associated reactions) were reported during or after infusions. The patients' vital signs and physical examination remained stable, and patients' satisfaction was high.
Conclusions:
Our results suggest that shortening the agalsidase beta infusion time to 90 min is safe and feasible in stably treated adult patients with Fabry disease and no recent infusion-associated reactions.
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