Safety and Tolerability of a Shorter Agalsidase Beta Infusion Time in Patients with Classic or Later-Onset Fabry Disease.

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.