preprints.org > medicine and pharmacology > medicine and pharmacology > doi: 10.20944/preprints202409.1863.v1

Preprint Brief Report Version 1 Preserved in Portico This version is not peer-reviewed

Version 1 : Received: 23 September 2024 / Approved: 24 September 2024 / Online: 24 September 2024 (05:53:29 CEST)

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, multihour infusion regimen imposes a substantial burden on patients. Methods : We assessed the safety and feasibility of shortening the agalsidase beta infusion time to 90 minutes in adult patients with classic or later-onset Fabry disease in the absence of premedication. A total of 39 consecutive adult patients (agalsidase-naive: n=6; significant comorbidities: n=15) with no recent infusion-associated reactions underwent a total of 85 agalsidase beta infusions in our tertiary reference center for lysosomal diseases. Each infusion was administered at a constant rate (between 0.78 and 1.17 mg/min, depending on the patient’s body weight). Results : No adverse events of any type (including discomfort and infusion-associated reactions) were reported during or after infusions. The patients’ vital signs remained stable, and patient satisfaction was high. Seven patients requested even shorter infusions (50 to 60 minutes) under strict medical supervision. Conclusions : Our results suggest that shortening the agalsidase beta infusion time to 90 minutes is safe and feasible in stably treated adult patients with Fabry disease and no recent infusion-associated reactions.

Fabry disease; agalsidase beta; quality of life; reduced infusion time; safety; tolerability

Medicine and Pharmacology, Medicine and Pharmacology

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