Open AccessStepwise shortening of agalsidase beta infusion duration in Fabry disease: Clinical experience with infusion rate escalation protocol
Author(s) -
Riccio Eleonora,
Zanfardino Mario,
Franzese Monica,
Capuano Ivana,
Buonanno Pasquale,
Ferreri Lucia,
Amicone Maria,
Pisani Antonio
Publication year - 2021
Publication title -
molecular genetics and genomic medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.765
H-Index - 29
ISSN - 2324-9269
DOI - 10.1002/mgg3.1659
Subject(s) - tolerability , medicine , enzyme replacement therapy , fabry disease , anesthesia , urology , disease , adverse effect
Background Although enzyme replacement therapy with agalsidase beta resulted in a variety of clinical benefits, life‐long biweekly intravenous infusion may impact on patients’ quality of life. Moreover, regular infusions are time‐consuming: although a stepwise shortening of infusion duration is allowed up to a minimum of 1.5 hr, in most centers it remains ≥3 hr, and no data exists about the safety and tolerability of agalsidase beta administration at maximum tolerated infusion rate. Methods In this study, we reported our experience with a stepwise infusion rate escalation protocol developed in our center in a cohort of 53 Fabry patients (both already receiving and treatment‐naΪve), and explored factors predictive for the infusion rate increase tolerability. Results Fifty‐two patients (98%) reduced infusion duration ≤3 hr; of these, 38 (72%) even reached a duration ≤2 hr. We found a significant difference between the mean duration reached by already treated and naΪve patients ( p < .01). More severely affected patients (male patients and those with lower enzyme activity) received longer infusions for higher risk of infusion‐associated reactions (IARs). A significant correlation between anti‐agalsidase antibodies and IARs was found. Conclusion Our infusion rate escalation protocol is safe and could improve patient compliance, satisfaction and quality of life.