| Title | Burden of illness of Pompe disease in patients only receiving supportive care |
|---|---|
| Published in | Journal of Inherited Metabolic Disease, p.1-8. ISSN 0141-8955. |
| Author | Kanters, T.A. (Tim A.); Hagemans, M.L.C. (Marloes); Beek, van der N.A.M.E. (Nadine); Rutten, F.F.H. (Frans); Ploeg, van der A.T. (Ans); Hakkaart-van Roijen, van L. (Leona) |
| Date | 2011-04-18 |
| Type | article |
| Abstract | 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. |
| Publication | http://hdl.handle.net/1765/26477 |
| OpenURL | Search this publication in (your) library |
| Persistent Identifier | urn:NBN:nl:ui:15-1765/26477 |
| Metadata | XML |
| Repository | Erasmus University Rotterdam |
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