Home-Based Care for Patients with Lysosomal Storage Disease: Experiences in Argentina

• Autor(a) principal: Brunelli,Maria Victoria
• Data de Publicação: 2019
• Outros Autores: Rabhansl,Maria Margarita, Delacre,Clara, Dankert,Maria Magdalena, Cuevillas,Maria Victoria, Frias,Catalina Terán
• Tipo de documento: Artigo
• Idioma: eng
• Título da fonte: Journal of Inborn Errors of Metabolism and Screening
• Texto Completo: http://old.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942019000100302
• topic: enzyme replacement therapy; homecare; adherence; Gaucher disease; Fabry disease; Hunter syndrome

ABSTRACT Enzyme replacement therapy (ERT) is a long term treatment for patients who suffer from lysosomal storage disease. A transversal descriptive study was conducted to evaluate advantages and disadvantages of a home-based care program for patients with Gaucher, Fabry and Mucopolysaccharidosis II (MPS II) diseases. A survey among patients and nurses involved in healthcare delivery at home was utilized for this study. The adherence rate was 92.9% over the study period. Eighty six point nine percent chose to carry out the treatment at home and 88.5% felt that their quality of life had improved. Additional advantages reported were: comfort (77%), treatment adjustment to daily activities (69%) and flexibility (58%). Disadvantages expressed were: lack of confidence with the health care provider at home (1.6%) and a shortage of disposable materials available (1.6%). The main benefits of home-based treatment were the high treatment adherence and the improvement in quality of life.