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Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe

Péntek, Márta; Gulácsi, László; Brodszky, Valentin; Baji, Petra; Boncz, Imre; Pogány, Gábor; López-Bastida, Julio; Linertová, Renata; Oliva-Moreno, Juan and Serrano-Aguilar, Pedro, et al. (2016) In European Journal of Health Economics 17. p.89-98
Abstract

Objectives: To assess the health-related quality of life (HRQOL) of patients with mucopolysaccharidosis (MPS) and their caregivers and to quantify the disease-related costs from a societal perspective. Methods: In the context of a multi-country study of rare diseases (BURQOL-RD project), a cross-sectional survey was performed among MPS patients in seven European countries. Data on demographic characteristics, health resource utilization, informal care, and loss of labor productivity were collected. The EQ-5D, Barthel index (BI), and Zarit burden interview (ZBI) questionnaires were used to assess patients’ and their informal caregivers’ quality of life, patients’ functional ability, and caregivers’ burden, respectively. Results:... (More)

Objectives: To assess the health-related quality of life (HRQOL) of patients with mucopolysaccharidosis (MPS) and their caregivers and to quantify the disease-related costs from a societal perspective. Methods: In the context of a multi-country study of rare diseases (BURQOL-RD project), a cross-sectional survey was performed among MPS patients in seven European countries. Data on demographic characteristics, health resource utilization, informal care, and loss of labor productivity were collected. The EQ-5D, Barthel index (BI), and Zarit burden interview (ZBI) questionnaires were used to assess patients’ and their informal caregivers’ quality of life, patients’ functional ability, and caregivers’ burden, respectively. Results: Altogether, 120 patients (children 62 %, females 40 %) and 66 caregivers completed the questionnaire. Patients’ mean age was 16.5 years and median age at diagnosis was 3 years. Adult patients’ average EQ-5D and EQ VAS scores varied across countries from 0.13 to 0.43 and 30.0 to 62.2, respectively, mean BI was 46.7, and ZBI was 32.7. Mean informal care time was 51.3 h/week. The mean total annual cost per patient (reference year 2012) was €24,520 in Hungary, €25,993 in France, €84,921 in Italy, €94,384 in Spain, and €209,420 in Germany. Costs are also shown to differ between children and adults. Direct costs accounted for most of the costs in all five countries (80, 100, 99, 98, and 93 %, respectively). Conclusions: MPS patients experience substantial loss of HRQOL and their families take a remarkable part in their care. Although utilization of health and social care resources varies significantly across countries, MPS incurs considerable societal costs in all the countries studied.

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keywords
Caregiver, Cost-of-illness, EQ-5D, Health-related quality of life, Mucopolysaccharidosis
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European Journal of Health Economics
volume
17
pages
10 pages
publisher
Springer
external identifiers
  • scopus:84964030231
  • wos:000380717200009
ISSN
1618-7598
DOI
10.1007/s10198-016-0787-0
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English
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yes
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494f4a32-7803-4556-9a4d-84c2eaefaa3e
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2016-06-20 15:44:13
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2017-10-16 12:28:46
@article{494f4a32-7803-4556-9a4d-84c2eaefaa3e,
  abstract     = {<p>Objectives: To assess the health-related quality of life (HRQOL) of patients with mucopolysaccharidosis (MPS) and their caregivers and to quantify the disease-related costs from a societal perspective. Methods: In the context of a multi-country study of rare diseases (BURQOL-RD project), a cross-sectional survey was performed among MPS patients in seven European countries. Data on demographic characteristics, health resource utilization, informal care, and loss of labor productivity were collected. The EQ-5D, Barthel index (BI), and Zarit burden interview (ZBI) questionnaires were used to assess patients’ and their informal caregivers’ quality of life, patients’ functional ability, and caregivers’ burden, respectively. Results: Altogether, 120 patients (children 62 %, females 40 %) and 66 caregivers completed the questionnaire. Patients’ mean age was 16.5 years and median age at diagnosis was 3 years. Adult patients’ average EQ-5D and EQ VAS scores varied across countries from 0.13 to 0.43 and 30.0 to 62.2, respectively, mean BI was 46.7, and ZBI was 32.7. Mean informal care time was 51.3 h/week. The mean total annual cost per patient (reference year 2012) was €24,520 in Hungary, €25,993 in France, €84,921 in Italy, €94,384 in Spain, and €209,420 in Germany. Costs are also shown to differ between children and adults. Direct costs accounted for most of the costs in all five countries (80, 100, 99, 98, and 93 %, respectively). Conclusions: MPS patients experience substantial loss of HRQOL and their families take a remarkable part in their care. Although utilization of health and social care resources varies significantly across countries, MPS incurs considerable societal costs in all the countries studied.</p>},
  author       = {Péntek, Márta and Gulácsi, László and Brodszky, Valentin and Baji, Petra and Boncz, Imre and Pogány, Gábor and López-Bastida, Julio and Linertová, Renata and Oliva-Moreno, Juan and Serrano-Aguilar, Pedro and Posada-de-la-Paz, Manuel and Taruscio, Domenica and Iskrov, Georgi and Schieppati, Arrigo and von der Schulenburg, Johann Matthias Graf and Kanavos, Panos and Chevreul, Karine and Persson, Ulf and Fattore, Giovanni},
  issn         = {1618-7598},
  keyword      = {Caregiver,Cost-of-illness,EQ-5D,Health-related quality of life,Mucopolysaccharidosis},
  language     = {eng},
  month        = {04},
  pages        = {89--98},
  publisher    = {Springer},
  series       = {European Journal of Health Economics},
  title        = {Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe},
  url          = {http://dx.doi.org/10.1007/s10198-016-0787-0},
  volume       = {17},
  year         = {2016},
}