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

Márta Péntek, László Gulácsi, Valentin Brodszky, Petra Baji, Imre Boncz, Gábor Pogány, Julio López-Bastida, Renata Linertová, Juan Oliva-Moreno, Pedro Serrano-Aguilar, Manuel Posada-de-la-Paz, Domenica Taruscio, Georgi Iskrov, Arrigo Schieppati, Johann Matthias Graf von der Schulenburg, Panos Kanavos, Karine Chevreul, Ulf Persson, Giovanni Fattore, Research Network Burqol-Rd Research Network

Research output: Contribution to journalArticle

8 Citations (Scopus)

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: 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.

Original languageEnglish
Pages (from-to)1-10
Number of pages10
JournalEuropean Journal of Health Economics
DOIs
Publication statusAccepted/In press - Apr 9 2016

Fingerprint

Mucopolysaccharidoses
Caregivers
Economics
Quality of Life
Costs and Cost Analysis
Patient Care
Patient Acceptance of Health Care
Interviews
Economic cost
Health-related quality of life
Cost of Illness
Aptitude
Hungary
Health Resources
Rare Diseases
Spain
Italy
France
Germany
Costs

Keywords

  • Caregiver
  • Cost-of-illness
  • EQ-5D
  • Health-related quality of life
  • Mucopolysaccharidosis

ASJC Scopus subject areas

  • Health Policy
  • Economics, Econometrics and Finance (miscellaneous)

Cite this

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; Serrano-Aguilar, Pedro; Posada-de-la-Paz, Manuel; Taruscio, Domenica; Iskrov, Georgi; Schieppati, Arrigo; von der Schulenburg, Johann Matthias Graf; Kanavos, Panos; Chevreul, Karine; Persson, Ulf; Fattore, Giovanni; Burqol-Rd Research Network, Research Network.

In: European Journal of Health Economics, 09.04.2016, p. 1-10.

Research output: Contribution to journalArticle

Péntek, M, Gulácsi, L, Brodszky, V, Baji, P, Boncz, I, Pogány, G, López-Bastida, J, Linertová, R, Oliva-Moreno, J, Serrano-Aguilar, P, Posada-de-la-Paz, M, Taruscio, D, Iskrov, G, Schieppati, A, von der Schulenburg, JMG, Kanavos, P, Chevreul, K, Persson, U, Fattore, G & Burqol-Rd Research Network, RN 2016, 'Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe', European Journal of Health Economics, pp. 1-10. https://doi.org/10.1007/s10198-016-0787-0
Péntek, Márta ; Gulácsi, László ; Brodszky, Valentin ; Baji, Petra ; Boncz, Imre ; Pogány, Gábor ; López-Bastida, Julio ; Linertová, Renata ; Oliva-Moreno, Juan ; Serrano-Aguilar, Pedro ; Posada-de-la-Paz, Manuel ; Taruscio, Domenica ; Iskrov, Georgi ; Schieppati, Arrigo ; von der Schulenburg, Johann Matthias Graf ; Kanavos, Panos ; Chevreul, Karine ; Persson, Ulf ; Fattore, Giovanni ; Burqol-Rd Research Network, Research Network. / Social/economic costs and health-related quality of life of mucopolysaccharidosis patients and their caregivers in Europe. In: European Journal of Health Economics. 2016 ; pp. 1-10.
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AU - Péntek, Márta

AU - Gulácsi, László

AU - Brodszky, Valentin

AU - Baji, Petra

AU - Boncz, Imre

AU - Pogány, Gábor

AU - López-Bastida, Julio

AU - Linertová, Renata

AU - Oliva-Moreno, Juan

AU - Serrano-Aguilar, Pedro

AU - Posada-de-la-Paz, Manuel

AU - Taruscio, Domenica

AU - Iskrov, Georgi

AU - Schieppati, Arrigo

AU - von der Schulenburg, Johann Matthias Graf

AU - Kanavos, Panos

AU - Chevreul, Karine

AU - Persson, Ulf

AU - Fattore, Giovanni

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