Health-Related Quality of Life in Patients Living with Wilson Disease in Spain : A Cross-Sectional Observational Study
Wilson disease (WD) is a rare copper metabolism disorder caused by mutations in the ATP7B gene. It usually affects young individuals and can produce hepatic and/or neurological involvement, potentially affecting health-related quality of life (HRQoL). We assessed HRQoL in a cohort of Spanish patients with WD and evaluated disease impact on several domains of patients' lives, treatment adherence, drug preference and satisfaction, and healthcare resource utilisation in a cross-sectional, retrospective, multicentric, observational study. A total of 102 patients were included: 81.4% presented isolated liver involvement (group H) and 18.6% presented neurological or mixed involvement (group EH). Up to 30% of patients reported a deteriorated emotional status with anxiety and depression, which was greater in the EH subgroup; the use of neuropsychiatric drugs was high. Over 70% of the patients were satisfied with their current treatment but complained about taking too many pills, stating they would consider switching to another more patient-friendly treatment if available. The Simplified Medication Adherence Questionnaire revealed only 22.5% of patients were fully adherent to therapy, suggesting that alternative therapies are needed. This real-world study, even though is highly enriched with hepatic patients and mild disease, shows that WD impacts patients' HRQoL, especially in the emotional domain.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:12 |
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| Enthalten in: |
Journal of clinical medicine - 12(2023), 14 vom: 21. Juli |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Mariño, Zoe [VerfasserIn] |
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| Links: |
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| Themen: |
Adherence |
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| Anmerkungen: |
Date Revised 01.08.2023 published: Electronic Citation Status PubMed-not-MEDLINE |
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| doi: |
10.3390/jcm12144823 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| 520 | |a Wilson disease (WD) is a rare copper metabolism disorder caused by mutations in the ATP7B gene. It usually affects young individuals and can produce hepatic and/or neurological involvement, potentially affecting health-related quality of life (HRQoL). We assessed HRQoL in a cohort of Spanish patients with WD and evaluated disease impact on several domains of patients' lives, treatment adherence, drug preference and satisfaction, and healthcare resource utilisation in a cross-sectional, retrospective, multicentric, observational study. A total of 102 patients were included: 81.4% presented isolated liver involvement (group H) and 18.6% presented neurological or mixed involvement (group EH). Up to 30% of patients reported a deteriorated emotional status with anxiety and depression, which was greater in the EH subgroup; the use of neuropsychiatric drugs was high. Over 70% of the patients were satisfied with their current treatment but complained about taking too many pills, stating they would consider switching to another more patient-friendly treatment if available. The Simplified Medication Adherence Questionnaire revealed only 22.5% of patients were fully adherent to therapy, suggesting that alternative therapies are needed. This real-world study, even though is highly enriched with hepatic patients and mild disease, shows that WD impacts patients' HRQoL, especially in the emotional domain | ||
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| 700 | 1 | |a Miralpeix, Anna |e verfasserin |4 aut | |
| 700 | 1 | |a Sastre, Isabel |e verfasserin |4 aut | |
| 700 | 1 | |a Reyes-Domínguez, Ana |e verfasserin |4 aut | |
| 700 | 1 | |a Castillo, Pilar |e verfasserin |4 aut | |
| 700 | 1 | |a García-Solà, Clàudia |e verfasserin |4 aut | |
| 700 | 1 | |a Bono, Ariadna |e verfasserin |4 aut | |
| 700 | 1 | |a Romero, Miriam |e verfasserin |4 aut | |
| 700 | 1 | |a Pérez-Sádaba, Francisco Javier |e verfasserin |4 aut | |
| 700 | 1 | |a Aceituno, Susana |e verfasserin |4 aut | |
| 700 | 1 | |a Anguera, Anna |e verfasserin |4 aut | |
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