scholarly journals Is home-based therapy in Fabry disease the answer to compelling patients’ needs during the COVID-19 pandemic? Survey results from the Polish FD Collaborative Group

2021 ◽  
Vol 30 (4) ◽  
pp. 449-454
Author(s):  
Mariusz Kusztal ◽  
Mariusz Kłopotowski ◽  
Stanisława Bazan-Socha ◽  
Beata Błażejewska-Hyżorek ◽  
Krzysztof Pawlaczyk ◽  
...  
Author(s):  
Michał Nowicki ◽  
Stanisława Bazan-Socha ◽  
Mariusz Kłopotowski ◽  
Beata Błażejewska-Hyżorek ◽  
Mariusz Kusztal ◽  
...  

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.


Medicine ◽  
2020 ◽  
Vol 99 (47) ◽  
pp. e23296
Author(s):  
Chong Pui Kei ◽  
Nor Azlin Mohd Nordin ◽  
Aznida Firzah Abdul Aziz

2021 ◽  
Vol 36 (Supplement_1) ◽  
Author(s):  
Mevlut Tamer Dincer ◽  
Cebrail Karaca ◽  
Betul Sarac ◽  
Saffa Ahmadzada ◽  
Alev Bakir ◽  
...  

Abstract Background and Aims Fabry disease is a rare metabolic disorder, lifelong enzyme replacement therapy with recombinant human alpha-galactosidase A (agalsidase) constituted the cornerstone of disease-specific therapy. COVID-19 pandemic and epidemic control measures including lockdowns impaired access to health care services. We examined the effect of COVID-19 pandemic and lockdown measures on mood status and management of Fabry disease patients. Method We conducted a cross-sectional study between October 2020 and December 2020. We used the Hospital Anxiety and Depression Scale (HADS) to evaluate the mood statuses of FD patients and the Morisky Medication Adherence Scale (MMAS-4) to assess patient adherence. We also examined age and sex-matched control group to compare mood status. Results A total of 68 (Male 48.5 %, mean age 37.0) FD patients were under regular follow-up in our institution, 59 of those patients were taking ERT every other week. Two of our patients had reported having a COVID-19 infection, and both of them recovered. 25 patients reported to miss an ERT for a median of one dose, 16 of these 25 patients have reported that they did not come to the hospital because of infection fear. Half of the patients had adopted home-based infusion; they arranged a nurse for home-based infusion therapy by their own means. According to MMAS-4 FD patients had good adherence to their therapy (Median score 0, range 0-2).  Mood status of FD patients and controls are shown in Table 1. Both HADS depression and anxiety scores were higher in the control group compared to FD patients. Additionally, abnormal scores were more prevalent for HADS depression scores in controls (Figure 1). Conclusion We found that the mood status of FD patients was better than the control group. Traumatic growth may be an important factor to explain this finding. Their adherence to therapy was good. Home-based therapy was the preferred method by the patients. Government-supported home therapy programs might be beneficial for FD patients to increase adherence to the therapy.


Author(s):  
Fiona Coupar ◽  
Alex Pollock ◽  
Lynn A Legg ◽  
Catherine Sackley ◽  
Paulette van Vliet

2020 ◽  
Vol 106 ◽  
pp. 17-23
Author(s):  
Harvey S. Singer ◽  
Shelley McDermott ◽  
Lisa Ferenc ◽  
Mathew Specht ◽  
E. Mark Mahone

2005 ◽  
Vol 6 (3) ◽  
pp. 227-241
Author(s):  
Lorenzo Pradelli

Sodium parnaparin is a low molecular weight heparin (LMWH). The introduction of this drug class has represented a medical advancement in the prevention and therapy of thromboembolic pathologies, as they maintain the same efficacy of unfractionated heparin, but with simplified dosing regimens and reduced side effects. Parnaparin has demonstrated its thromboprophylactic efficacy on both high- and moderate risk surgical patients, besides resulting effective in treating established deep vein thromboses and thrombosis-associated phlebopathies. Alongside these clinical advantages, parnaparin and other LMWHs allow outpatient or home-based therapy of a large number of subjects that should otherwise be treated in hospital, with important savings of health resources and enhanced quality of life for the patients. In Italy, parnaparin drug acquisition cost is among the lowest in its class for many applications, permitting marginal savings in health costs at the same efficacy level. In summary, the use of parnaparin in thrombotic pathologies has very good efficacy and safety profiles, and has positive clinical and economical outcomes for patients, health system and society as a whole.


Sign in / Sign up

Export Citation Format

Share Document