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Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic

Objective: Patients with Lysosomal disorders (LSDs) are treated with regular infusions of enzyme replacement therapy (ERT). During the COVID-19 pandemic, home treatment was permitted. This study aimed at monitoring the patients’ compliance with home therapy and its effects on physical, psychological...

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Autores principales: Fiumara, Agata, Lanzafame, Giuseppina, Sapuppo, Annamaria, Arena, Alessia, Cirnigliaro, Lara, Barone, Rita
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10137846/
https://www.ncbi.nlm.nih.gov/pubmed/37108010
http://dx.doi.org/10.3390/healthcare11081176
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author Fiumara, Agata
Lanzafame, Giuseppina
Sapuppo, Annamaria
Arena, Alessia
Cirnigliaro, Lara
Barone, Rita
author_facet Fiumara, Agata
Lanzafame, Giuseppina
Sapuppo, Annamaria
Arena, Alessia
Cirnigliaro, Lara
Barone, Rita
author_sort Fiumara, Agata
collection PubMed
description Objective: Patients with Lysosomal disorders (LSDs) are treated with regular infusions of enzyme replacement therapy (ERT). During the COVID-19 pandemic, home treatment was permitted. This study aimed at monitoring the patients’ compliance with home therapy and its effects on physical, psychological, and relational issues. Moreover, we also tested the possible impact of home therapy on familial relationships and contacts with the referral hospital. Materials and Methods: Thirteen patients with Pompe disease (N = 8) and MPS (N = 5) were tested through an online questionnaire designed to assess their level of appreciation and satisfaction with home therapy and their feelings about the referral centre and psychological support provided. Results: Most of the patients (84%) stressed the positive impact of home therapy. All patients described a significant reduction in stressful conditions associated with the need to attend the hospital every week or two. Conclusions: Home ERT leads to a clear improvement in “daily life skills”, as represented in our by sample by positive feelings, better emotional self-control, and an increased ability to understand the feelings of relatives. Our data underline the paramount positive effect home ERT has on both patients and their families.
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spelling pubmed-101378462023-04-28 Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic Fiumara, Agata Lanzafame, Giuseppina Sapuppo, Annamaria Arena, Alessia Cirnigliaro, Lara Barone, Rita Healthcare (Basel) Communication Objective: Patients with Lysosomal disorders (LSDs) are treated with regular infusions of enzyme replacement therapy (ERT). During the COVID-19 pandemic, home treatment was permitted. This study aimed at monitoring the patients’ compliance with home therapy and its effects on physical, psychological, and relational issues. Moreover, we also tested the possible impact of home therapy on familial relationships and contacts with the referral hospital. Materials and Methods: Thirteen patients with Pompe disease (N = 8) and MPS (N = 5) were tested through an online questionnaire designed to assess their level of appreciation and satisfaction with home therapy and their feelings about the referral centre and psychological support provided. Results: Most of the patients (84%) stressed the positive impact of home therapy. All patients described a significant reduction in stressful conditions associated with the need to attend the hospital every week or two. Conclusions: Home ERT leads to a clear improvement in “daily life skills”, as represented in our by sample by positive feelings, better emotional self-control, and an increased ability to understand the feelings of relatives. Our data underline the paramount positive effect home ERT has on both patients and their families. MDPI 2023-04-19 /pmc/articles/PMC10137846/ /pubmed/37108010 http://dx.doi.org/10.3390/healthcare11081176 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Communication
Fiumara, Agata
Lanzafame, Giuseppina
Sapuppo, Annamaria
Arena, Alessia
Cirnigliaro, Lara
Barone, Rita
Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic
title Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic
title_full Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic
title_fullStr Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic
title_full_unstemmed Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic
title_short Positive Impact of Home ERT for Mucopolysaccharidoses and Pompe Disease: The Lesson Learnt from the COVID-19 Pandemic
title_sort positive impact of home ert for mucopolysaccharidoses and pompe disease: the lesson learnt from the covid-19 pandemic
topic Communication
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10137846/
https://www.ncbi.nlm.nih.gov/pubmed/37108010
http://dx.doi.org/10.3390/healthcare11081176
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