BACKGROUND: Enzyme replacement therapy for Fabry disease, consisting of biweekly infusions, interferes daily life. Home treatment proved beneficial. We evaluated a previously reported home treatment algorithm aiming to shorten the period of in-hospital infusions, while ascertaining patient safety. METHODS: Retrospective analysis on clinical records of treated Fabry patients. Potentially predictive factors for infusion associated reactions (IARs) were studied: agalsidase antibodies, agalsidase product and dose, FOS-SSI scores, and GLA activity and mutation. A questionnaire evaluated patient satisfaction and compliance. RESULTS: Seventy-nine patients were included (41 males, 46% agalsidase antibody positive (AB+)). 85% received home treatment. Home treatment complications were erroneous fast infusion rates (n=4) causing IARs and, rarely, venous access problems. The single SAE was unrelated to home treatment. IgG antibody status was significantly associated with IARs (89% vs. 26% p-value<0.01). Negative antibody status did not preclude IARs. Except for three AB+ patients, all first IARs occurred within 13 infusions. IARs occurred more frequently in patients using agalsidase beta 1.0 mg/kg/eow than agalsidase alpha or beta 0.2 mg/kg/eow, but the time to first IAR did not differ between groups. Four AB+ males experienced IARs after a dose increase. Compliance between home and in-hospital treatment was similar. Most patients preferred home treatment. CONCLUSION: In this study home therapy for Fabry disease was safe and improved patient satisfaction. We propose a revised algorithm which allows safe home-treatment in all male patients after 13 instead of 26 infusions, irrespective of ERT preparation or dose. Furthermore, AB+ patients with dosage increase may experience new or increased IARs, necessitating in-hospital observations.
BACKGROUND: Enzyme replacement therapy for Fabry disease, consisting of biweekly infusions, interferes daily life. Home treatment proved beneficial. We evaluated a previously reported home treatment algorithm aiming to shorten the period of in-hospital infusions, while ascertaining patient safety. METHODS: Retrospective analysis on clinical records of treated Fabry patients. Potentially predictive factors for infusion associated reactions (IARs) were studied: agalsidase antibodies, agalsidase product and dose, FOS-SSI scores, and GLA activity and mutation. A questionnaire evaluated patient satisfaction and compliance. RESULTS: Seventy-nine patients were included (41 males, 46% agalsidase antibody positive (AB+)). 85% received home treatment. Home treatment complications were erroneous fast infusion rates (n=4) causing IARs and, rarely, venous access problems. The single SAE was unrelated to home treatment. IgG antibody status was significantly associated with IARs (89% vs. 26% p-value<0.01). Negative antibody status did not preclude IARs. Except for three AB+ patients, all first IARs occurred within 13 infusions. IARs occurred more frequently in patients using agalsidase beta 1.0 mg/kg/eow than agalsidase alpha or beta 0.2 mg/kg/eow, but the time to first IAR did not differ between groups. Four AB+ males experienced IARs after a dose increase. Compliance between home and in-hospital treatment was similar. Most patients preferred home treatment. CONCLUSION: In this study home therapy for Fabry disease was safe and improved patient satisfaction. We propose a revised algorithm which allows safe home-treatment in all male patients after 13 instead of 26 infusions, irrespective of ERT preparation or dose. Furthermore, AB+ patients with dosage increase may experience new or increased IARs, necessitating in-hospital observations.
Authors: Michał Nowicki; Stanisława Bazan-Socha; Mariusz Kłopotowski; Beata Błażejewska-Hyżorek; Mariusz Kusztal; Krzysztof Pawlaczyk; Jarosław Sławek; Andrzej Oko; Zofia Oko-Sarnowska Journal: Int J Environ Res Public Health Date: 2021-08-04 Impact factor: 3.390
Authors: Malte Lenders; Leon Paul Neußer; Michael Rudnicki; Peter Nordbeck; Sima Canaan-Kühl; Albina Nowak; Markus Cybulla; Boris Schmitz; Jan Lukas; Christoph Wanner; Stefan-Martin Brand; Eva Brand Journal: J Am Soc Nephrol Date: 2018-11-01 Impact factor: 10.121
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