The patients` perspective on home-based infusion: A longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy
Background: Lysosomal storage diseases (LSDs), metabolic disorders resulting in build-up of endogenous waste and progressive organ damage, can be treated with intravenous enzyme replacement therapy (ERT). ERT can be administered either in specialized clinics, at a physician's office or in the h...
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| Format: | Article |
| Language: | English |
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Elsevier
2023-06-01
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| Series: | Molecular Genetics and Metabolism Reports |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2214426923000174 |
| _version_ | 1797822045283680256 |
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| author | Ria Heinrich Franziska Claus Tonio Schoenfelder |
| author_facet | Ria Heinrich Franziska Claus Tonio Schoenfelder |
| author_sort | Ria Heinrich |
| collection | DOAJ |
| description | Background: Lysosomal storage diseases (LSDs), metabolic disorders resulting in build-up of endogenous waste and progressive organ damage, can be treated with intravenous enzyme replacement therapy (ERT). ERT can be administered either in specialized clinics, at a physician's office or in the home care setting. Legislative goals in Germany strive to shift to more outpatient care while maintaining treatment objectives. This study investigates the patient perspective on home-based ERT in terms of acceptance, safety, and treatment satisfaction in LSD patients. Methods: This was a longitudinal observational study, carried out under real-world conditions in patients' home environment, covering 30 months from January 2019 to June 2021. Patients with LSDs who were deemed suitable for home-based ERT by their physicians were recruited to the study. Patients were interviewed before the start of the first home-based ERT and then at regular intervals thereafter using standardized questionnaires. Results: Data from 30 patients were analyzed: 18 with Fabry disease, 5 with Gaucher disease, 6 with Pompe disease and 1 with Mucopolysaccharidosis type I (MPS I). Age ranged from 8 to 77 years (mean age 40). The reported average waiting time prior to infusion of more than half an hour decreased from 30% of the patients affected at baseline to 5% across all follow-up time points. All patients felt adequately informed about home-based ERT throughout follow-ups and reported that they would choose home-based ERT again. At almost each time point, patients indicated that home-based ERT had improved their ability to cope with the disease. All but one patient indicated feeling safe at each follow-up time point. Compared to 36.7% at baseline, only 6.9% of patients reported a need for improvement in their care after 6 months of home-based ERT. Mean treatment satisfaction increased by approximately 1.6 scale points after 6 months of home-based ERT compared to baseline, and by another 0.2 scale points after 18 months. In terms of quality of care, all but one patient rated home-based ERT as an equivalent alternative throughout follow-ups. Patients would recommend home-based ERT to other suitable LSD-patients. Conclusion: Home-based ERT increases patients' treatment satisfaction, and patients perceive the quality of care as an equivalent alternative, compared to ERT in a center, clinic, or at a physician's office. |
| first_indexed | 2024-03-13T10:03:03Z |
| format | Article |
| id | doaj.art-45dfbaa0f31c4c77a20fc89513604be3 |
| institution | Directory Open Access Journal |
| issn | 2214-4269 |
| language | English |
| last_indexed | 2024-03-13T10:03:03Z |
| publishDate | 2023-06-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Molecular Genetics and Metabolism Reports |
| spelling | doaj.art-45dfbaa0f31c4c77a20fc89513604be32023-05-23T04:21:49ZengElsevierMolecular Genetics and Metabolism Reports2214-42692023-06-0135100971The patients` perspective on home-based infusion: A longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapyRia Heinrich0Franziska Claus1Tonio Schoenfelder2Scientific Institute for Health Economics and Health System Research [Wissenschaftliches Institut für Gesundheitsökonomie und Gesundheitssystemforschung] (WIG2), Markt 8, Leipzig 04109, GermanyScientific Institute for Health Economics and Health System Research [Wissenschaftliches Institut für Gesundheitsökonomie und Gesundheitssystemforschung] (WIG2), Markt 8, Leipzig 04109, GermanyScientific Institute for Health Economics and Health System Research [Wissenschaftliches Institut für Gesundheitsökonomie und Gesundheitssystemforschung] (WIG2), Markt 8, Leipzig 04109, Germany; Technische Universität Dresden, Chair of Health Sciences - Public Health, Löscherstraße 18, Dresden 01309, Germany; Corresponding author at: WIG2 GmbH, Markt 8, Leipzig 04109, Germany.Background: Lysosomal storage diseases (LSDs), metabolic disorders resulting in build-up of endogenous waste and progressive organ damage, can be treated with intravenous enzyme replacement therapy (ERT). ERT can be administered either in specialized clinics, at a physician's office or in the home care setting. Legislative goals in Germany strive to shift to more outpatient care while maintaining treatment objectives. This study investigates the patient perspective on home-based ERT in terms of acceptance, safety, and treatment satisfaction in LSD patients. Methods: This was a longitudinal observational study, carried out under real-world conditions in patients' home environment, covering 30 months from January 2019 to June 2021. Patients with LSDs who were deemed suitable for home-based ERT by their physicians were recruited to the study. Patients were interviewed before the start of the first home-based ERT and then at regular intervals thereafter using standardized questionnaires. Results: Data from 30 patients were analyzed: 18 with Fabry disease, 5 with Gaucher disease, 6 with Pompe disease and 1 with Mucopolysaccharidosis type I (MPS I). Age ranged from 8 to 77 years (mean age 40). The reported average waiting time prior to infusion of more than half an hour decreased from 30% of the patients affected at baseline to 5% across all follow-up time points. All patients felt adequately informed about home-based ERT throughout follow-ups and reported that they would choose home-based ERT again. At almost each time point, patients indicated that home-based ERT had improved their ability to cope with the disease. All but one patient indicated feeling safe at each follow-up time point. Compared to 36.7% at baseline, only 6.9% of patients reported a need for improvement in their care after 6 months of home-based ERT. Mean treatment satisfaction increased by approximately 1.6 scale points after 6 months of home-based ERT compared to baseline, and by another 0.2 scale points after 18 months. In terms of quality of care, all but one patient rated home-based ERT as an equivalent alternative throughout follow-ups. Patients would recommend home-based ERT to other suitable LSD-patients. Conclusion: Home-based ERT increases patients' treatment satisfaction, and patients perceive the quality of care as an equivalent alternative, compared to ERT in a center, clinic, or at a physician's office.http://www.sciencedirect.com/science/article/pii/S2214426923000174Lysosomal storage diseaseEnzyme replacement therapyHome infusionGermanyPatient satisfaction |
| spellingShingle | Ria Heinrich Franziska Claus Tonio Schoenfelder The patients` perspective on home-based infusion: A longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy Molecular Genetics and Metabolism Reports Lysosomal storage disease Enzyme replacement therapy Home infusion Germany Patient satisfaction |
| title | The patients` perspective on home-based infusion: A longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy |
| title_full | The patients` perspective on home-based infusion: A longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy |
| title_fullStr | The patients` perspective on home-based infusion: A longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy |
| title_full_unstemmed | The patients` perspective on home-based infusion: A longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy |
| title_short | The patients` perspective on home-based infusion: A longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy |
| title_sort | patients perspective on home based infusion a longitudinal observational study in the german healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy |
| topic | Lysosomal storage disease Enzyme replacement therapy Home infusion Germany Patient satisfaction |
| url | http://www.sciencedirect.com/science/article/pii/S2214426923000174 |
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