Burden of illness of Pompe disease in patients only receiving supportive care.
J Inherit Metab Dis
; 34(5): 1045-52, 2011 Oct.
Article
en En
| MEDLINE
| ID: mdl-21499718
BACKGROUND: Pompe disease is an orphan disease for which enzyme replacement therapy (ERT) recently became available. This study aims to estimate all relevant aspects of burden of illness--societal costs, use of home care and informal care, productivity losses, and losses in health-related quality of life (HRQoL)--for adult Pompe patients only receiving supportive care. METHODS: We collected data on all relevant aspects of burden of illness via a questionnaire. We applied a societal perspective in calculating costs. The EQ-5D was used to estimate HRQoL. RESULTS: Eighty adult patients (87% of the total Dutch adult Pompe population) completed a questionnaire. Disease severity ranged from mild to severe. Total annual costs were estimated at 22,475 (range 0-169,539) per adult Pompe patient. Patients on average received 8 h of home care and 19 h of informal care per week. Eighty-five percent of the patients received informal care from one or more caregivers; 40% had stopped working due to their disease; another 20% had reduced their working hours. HRQoL for Pompe patients who only received supportive care was estimated at 0.72, 17% lower than the Dutch population at large. CONCLUSIONS: Adult Pompe disease is associated with a considerable burden of illness at both the societal and patient levels. The disease leads to substantial costs and dependency on medical devices, home care, and informal care, and has a high impact on the patient's social network. In addition, patients are limited in their ability to work and have significantly reduced HRQoL.
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Cuidados Paliativos
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Enfermedad del Almacenamiento de Glucógeno Tipo II
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Costo de Enfermedad
Tipo de estudio:
Evaluation_studies
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Observational_studies
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Prognostic_studies
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Qualitative_research
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Risk_factors_studies
Límite:
Adult
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Aged
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Female
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Humans
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Male
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Middle aged
Idioma:
En
Revista:
J Inherit Metab Dis
Año:
2011
Tipo del documento:
Article
País de afiliación:
Países Bajos