Stepwise Shortening Of Agalsidase Beta Infusion Duration In Fabry Disease: Clinical Experience With Infusion Rate Escalation Protocol

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-naive), 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 naive 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.