Eleonora Riccio, Mario Zanfardino, Monica Franzese, Ivana Capuano, Pasquale Buonanno, Lucia Ferreri, Maria Amicone, Antonio Pisani
Molecular genetics & genomic medicine 2021 MayAlthough 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. 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. 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. Our infusion rate escalation protocol is safe and could improve patient compliance, satisfaction and quality of life. © 2021 The Authors. Molecular Genetics & Genomic Medicine published by Wiley Periodicals LLC.
Eleonora Riccio, Mario Zanfardino, Monica Franzese, Ivana Capuano, Pasquale Buonanno, Lucia Ferreri, Maria Amicone, Antonio Pisani. Stepwise shortening of agalsidase beta infusion duration in Fabry disease: Clinical experience with infusion rate escalation protocol. Molecular genetics & genomic medicine. 2021 May;9(5):e1659
PMID: 33755336
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