Considerations for Home-Based Treatment of Fabry Disease in Poland during the COVID-19 Pandemic and Beyond

Michał Nowicki, Stanisława Bazan-Socha, Mariusz Kłopotowski, Beata Błażejewska-Hyżorek, Mariusz Kusztal, Krzysztof Pawlaczyk, Jarosław Sławek, Andrzej Oko and Zofia Oko-Sarnowska
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Michał Nowicki: Department of Nephrology, Hypertension, and Kidney Transplantation, Medical University of Łódź, 90-145 Łódź, Łódzkie, Poland
Stanisława Bazan-Socha: Department of Internal Medicine, Jagiellonian University Medical College, 30-688 Kraków, Małopolskie, Poland
Mariusz Kłopotowski: Department of Interventional Cardiology and Angiology, Institute of Cardiology, 04-628 Warsaw, Mazowieckie, Poland
Beata Błażejewska-Hyżorek: 2nd Department of Neurology, Institute of Psychiatry and Neurology, 02-957 Warsaw, Mazowieckie, Poland
Mariusz Kusztal: Department of Nephrology and Transplantation Medicine, Wrocław Medical University, 50-556 Wrocław, Dolnośląskie, Poland
Krzysztof Pawlaczyk: Department of Nephrology, University Hospital of Karol Marcinkowski in Zielona Góra, 65-046 Zielona Góra, Lubuskie, Poland
Jarosław Sławek: Division of Neurological and Psychiatric Nursing, Institute of Nursing and Midwifery, Medical University of Gdańsk, 80-462 Gdańsk, Pomorskie, Poland
Andrzej Oko: Department of Nephrology, Transplantology, and Internal Diseases, Poznań University of Medical Sciences, 60-355 Poznań, Wielkopolskie, Poland
Zofia Oko-Sarnowska: Department of Cardiology, Poznań University of Medical Sciences, 60-355 Poznań, Wielkopolskie, Poland

IJERPH, 2021, vol. 18, issue 16, 1-12

Abstract: Current therapy for Anderson–Fabry disease in Poland includes hospital or clinic-based intravenous enzyme replacement therapy with recombinant agalsidase alpha or beta, or oral pharmacological chaperone therapy with migalastat. Some countries around the world offer such treatment to patients in the comfort of their own homes. The 2020–2021 COVID-19 pandemic has pushed global healthcare providers to evolve their services so as to minimize the risk of COVID-19 exposure to both patients and providers; this has led to advances in telemedicine services and the increasing availability of at-home treatment for various procedures including parenteral drug administration. A total of 80% of surveyed Anderson–Fabry disease patients in Poland would prefer home-based treatment, which would be a safe and convenient alternative to clinic-based treatment if patient selection is based on our proposed algorithm. Our recommendations for home-based treatments appear feasible for the long term care of Anderson–Fabry disease patients during the COVID-19 pandemic and beyond. This may also serve as a basis for home-based treatment programs in other rare and ultra-rare genetic diseases.

Keywords: enzyme replacement therapy; Fabry disease; home-based treatment; telemedicine (search for similar items in EconPapers)
JEL-codes: I I1 I3 Q Q5 (search for similar items in EconPapers)
Date: 2021
References: View complete reference list from CitEc
Citations: View citations in EconPapers (1)

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