Skip to main content

Advertisement

SpringerLink
Log in

Comparison of EQ-5D and SF-6D utilities in Pompe disease

  • Published:
Quality of Life Research Aims and scope Submit manuscript

Abstract

Purpose

Comparative studies between Euroqol-5D (EQ-5D) and ShortForm 6D (SF-6D) utilities have been performed for a number of diseases, but not yet for orphan diseases. Pompe disease is an orphan disease with a prevalence of <5/10,000, characterized by impaired ambulatory and pulmonary functioning. We compared the psychometric properties of EQ-5D and SF-6D in patients with this disease and assessed their convergent validity, discriminative ability and sensitivity to change.

Methods

EQ-5D utilities and SF-6D utilities were computed using the UK value set. Dimensions and utilities of the two instruments were compared by correlation coefficients and descriptive statistics. We assessed whether EQ-5D and SF-6D were able to discriminate between different levels of severity and examined sensitivity to change for patients with multiple observations.

Results

Correlations between theoretically related dimensions of the EQ-5D and SF-6D were highly significant and were moderate to strong (range rho = 0.409–0.564). Utility values derived from the two instruments were similar (mean EQ-5D = 0.670; mean SF-6D = 0.699) and correlated strongly (rho = 0.591). Discriminative properties were somewhat better for EQ-5D; mean changes and effect sizes were better for SF-6D.

Conclusions

Overall, we conclude that both instruments appear to be equally appropriate with respect to assessing utilities in Pompe disease, but neither of them performed excellently. The descriptive system of the SF-6D describes health states for Pompe disease more accurately. EQ-5D showed better discriminative properties. The SF-6D performed better with respect to sensitivity to change.

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Fig. 1
Fig. 2

Similar content being viewed by others

References

  1. European Commission. (2000). Regulation (EC) no 141/2000 of the European parliament and of the council of 16 December 1999 on orphan medicinal products. Official Journal of the European Communities, L18/1.

  2. Haffner, M. E. (2006). Adopting orphan drugs—two dozen years of treating rare diseases. New England Journal of Medicine, 354, 445–447.

    Article  CAS  PubMed  Google Scholar 

  3. Schey, C., Milanova, T., & Hutchings, A. (2011). Estimating the budget impact of orphan medicines in Europe: 2010–2020. Orphanet Journal of Rare Diseases, 6, 62.

    Article  PubMed Central  PubMed  Google Scholar 

  4. Drummond, M. F., Wilson, D. A., Kanavos, P., Ubel, P., & Rovira, J. (2007). Assessing the economic challenges posed by orphan drugs. International Journal of Technology Assessment in Health Care, 23, 36–42.

    Article  PubMed  Google Scholar 

  5. van der Ploeg, A. T., & Reuser, A. J. J. (2008). Pompe’s disease. The Lancet, 372, 1342–1353.

    Article  Google Scholar 

  6. Ausems, M. G. E. M., Verbiest, J., Hermans, M. M. P., Kroos, M. A., Beemer, F. A., Wokke, J. H. J., et al. (1999). Frequency of glycogen storage disease type II in the Netherlands: Implications for diagnosis and genetic counselling. European Journal of Human Genetics, 7, 713–716.

    Article  CAS  PubMed  Google Scholar 

  7. Van der Beek, N. A. M. E., Van Capelle, C. I., Van der Velden-Van Etten, K. I., Hop, W. C. J., Van den Berg, B., Reuser, A. J. J., et al. (2011). Rate of progression and predictive factors for pulmonary outcome in children and adults with Pompe disease. Molecular Genetics and Metabolism, 104, 129–136.

    Article  PubMed  Google Scholar 

  8. Müller-Felber, W., Horvath, R., Gempel, K., Podskarbi, T., Shin, Y., Pongratz, D., et al. (2007). Late onset Pompe disease: Clinical and neurophysiological spectrum of 38 patients including long-term follow-up in 18 patients. Neuromuscular Disorders, 17, 698–706.

    Article  PubMed  Google Scholar 

  9. Van der Beek, N. A. M. E., De Vries, J. M., Hagemans, M. L., Hop, W. C., Kroos, M. A., Wokke, J. H., et al. (2012). Clinical features and predictors for disease natural progression in adults with Pompe disease: A nationwide prospective observational study. Orphanet Journal of Rare Diseases, 7, 88.

    Article  PubMed Central  PubMed  Google Scholar 

  10. Van der Beek, N. A. M. E., Hagemans, M. L. C., Reuser, A. J. J., Hop, W. C. J., van der Ploeg, A. T., Van Doorn, P. A., et al. (2009). Rate of disease progression during long-term follow-up of patients with late-onset Pompe disease. Neuromuscular Disorders, 19, 113–117.

    Article  PubMed  Google Scholar 

  11. Hagemans, M. L. C., Janssens, A., Winkel, L. P. F., Sieradzan, K. A., Reuser, A. J. J., Van Doorn, P. A., et al. (2004). Late-onset Pompe disease primarily affects quality of life in physical health domains. Neurology, 63, 1688–1692.

    Article  CAS  PubMed  Google Scholar 

  12. Kanters, T. A., Hagemans, M. L. C., Van der Beek, N. A. M. E., Rutten, F. F. H., van der Ploeg, A. T., & Hakkaart, L. (2011). Burden of illness of Pompe disease in patients only receiving supportive care. Journal of Inherited Metabolic Disease, 34, 1045–1052.

    Article  PubMed Central  PubMed  Google Scholar 

  13. Güngör, D., De Vries, J. M., Hop, W. C. J., Reuser, A. J. J., Van Doorn, P. A., van der Ploeg, A. T., et al. (2011). Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy. Orphanet Journal of Rare Diseases, 6, 34.

    Article  PubMed Central  PubMed  Google Scholar 

  14. Lamers, L. M., Bouwmans, C. A. M., Van Straten, A., Donker, M. C. H., & Hakkaart, L. (2006). Comparison of EQ-5D and SF-6D utilities in mental health patients. Health Economics, 15, 1229–1236.

    Article  CAS  PubMed  Google Scholar 

  15. Joore, M., Brunenberg, D., Nelemans, P., Wouters, E., Kuijpers, P., Honig, A., et al. (2010). The impact of differences in EQ-5D and SF-6D utility scores on the acceptability of cost-utility ratios: Results across five trial-based cost-utility studies. Value in Health, 13, 222–229.

    Article  PubMed  Google Scholar 

  16. Sach, T. H., Barton, G. R., Jenkinson, C., Doherty, M., Avery, A. J., & Muir, K. R. (2009). Comparing cost-utility estimates: Does the choice of EQ-5D or SF-6D matter? Medical Care, 47, 889–894.

    Article  PubMed  Google Scholar 

  17. Brooks, R. (1996). EuroQol: The current state of play. Health Policy, 37, 53–72.

    Article  CAS  PubMed  Google Scholar 

  18. Brazier, J., Roberts, J., & Deverill, M. (2002). The estimation of a preference-based measure of health from the SF-36. Journal of Health Economics, 21, 271–292.

    Article  PubMed  Google Scholar 

  19. Dolan, P. (1997). Modeling valuations for EuroQol health states. Medical Care, 35, 1095–1108.

    Article  CAS  PubMed  Google Scholar 

  20. Brazier, J., Tsuchiya, A., Roberts, J., & Busschbach, J. (2004). A comparison of the EQ-5D and the SF-6D across seven patient groups. Health Economics, 13, 873–884.

    Article  PubMed  Google Scholar 

  21. Stavem, K., Frøland, S. S., & Hellum, K. B. (2005). Comparison of preference-based utilities of the 15D, EQ-5D and SF-6D in patients with HIV/AIDS. Quality of Life Research, 14, 971–980.

    Article  PubMed  Google Scholar 

  22. Szende, A., Leidy, N. K., Ståhl, E., & Svensson, K. (2009). Estimating health utilities in patients with asthma and COPD: Evidence on the performance of EQ-5D and SF-6D. Quality of Life Research, 18, 267–272.

    Article  CAS  PubMed  Google Scholar 

  23. Marra, C. A., Woolcott, J. C., Kopec, J. A., Shojania, K., Offer, R., Brazier, J. E., et al. (2005). A comparison of generic, indirect utility measures (the HUI2, HUI3, SF-6D, and the EQ-5D) and disease-specific instruments (the RAQoL and the HAQ) in rheumatoid arthritis. Social Science and Medicine, 60, 1571–1582.

    Article  PubMed  Google Scholar 

  24. Fisk, J. D., Brown, M. G., Sketris, I. S., Metz, L. M., Murray, T. J., & Stadnyk, K. J. (2005). A comparison of health utility measures for the evaluation of multiple sclerosis treatments. Journal of Neurology, Neurosurgery and Psychiatry, 76, 58–63.

    Article  PubMed Central  CAS  PubMed  Google Scholar 

  25. Quanjer, P. H., Tammeling, G. J., Cotes, J. E., Pedersen, O. F., Peslin, R., & Yernault, J. C. (1993). Lung volumes and forced ventilatory flows. Report working party standardization of lung function tests, European community for steel and coal. official statement of the European respiratory society. European Respiratory Journal. Supplement, 16, 5–40.

    Article  CAS  Google Scholar 

  26. Medical Research Council. (1986). Aids to the examination of the peripheral nervous system. London: Tindall.

    Google Scholar 

  27. De Vries, J. M., Van der Beek, N. A. M. E., Hop, W. C. J., Karstens, F. P. J., Wokke, J. H., De Visser, M., et al. (2012). Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: An open-label single-center study. Orphanet Journal of Rare Diseases, 7, 73.

    Article  PubMed Central  PubMed  Google Scholar 

  28. Field, A. P. (2009). Discovering statistics using SPSS. London: SAGE publications Ltd.

    Google Scholar 

  29. Streiner, D. L., & Norman, G. R. (2008). Health measurement scales. Oxford: Oxford University Press.

    Book  Google Scholar 

  30. Ghislandi, S., Apolone, G., Garattini, L., & Ghislandi, I. (2002). Is EQ-5D a valid measure of HRQoL in patients with movement disorders? The European Journal of Health Economics, 3, 125–130.

    Article  CAS  PubMed  Google Scholar 

  31. Janssen, M. F., Birnie, E., Haagsma, J. A., & Bonsel, G. J. (2008). Comparing the standard EQ-5D three-level system with a five-level version. Value in Health, 11, 275–284.

    Article  PubMed  Google Scholar 

  32. McTaggart-Cowan, H. M., Marra, C. A., Yang, Y., Brazier, J. E., Kopec, J. A., FitzGerald, J. M., et al. (2008). The validity of generic and condition-specific preference-based instruments: The ability to discriminate asthma control status. Quality of Life Research, 17, 453–462.

    Article  PubMed  Google Scholar 

  33. Khanna, D., Furst, D. E., Wong, W. K., Tsevat, J., Clements, P. J., Park, G. S., et al. (2007). Reliability, validity, and minimally important differences of the SF-6D in systemic sclerosis. Quality of Life Research, 16, 1083–1092.

    Article  PubMed  Google Scholar 

  34. Lamers, L. M., McDonnell, J., Stalmeier, P. F. M., Krabbe, P. F. M., & Busschbach, J. J. V. (2006). The Dutch tariff: Results and arguments for an effective design for national EQ-5D valuation studies. Health Economics, 15, 1121–1132.

    Article  CAS  PubMed  Google Scholar 

Download references

Acknowledgments

This study was financially supported by the Netherlands Organization for Health Research and Development (ZonMw; grant no. 152001005). The authors would like to thank M.L.C. Hagemans for comments on earlier versions of the manuscript.

Author information

Authors and Affiliations

  1. Department of Health Policy and Management, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Burgemeester Oudlaan 50, P.O. Box 1738, 3000, DR, Rotterdam, The Netherlands

    T. A. Kanters, W. K. Redekop, M. P. M. H. Rutten-van Mölken & L. Hakkaart

  2. Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Dr Molewaterplein 60, 3015, GJ, Rotterdam, The Netherlands

    T. A. Kanters, M. E. Kruijshaar & A. T. van der Ploeg

  3. Division of Metabolic Diseases and Genetics, Department of Pediatrics, Erasmus MC University Medical Center Rotterdam, Dr Molewaterplein 60, 3015, GJ, Rotterdam, The Netherlands

    M. E. Kruijshaar & A. T. van der Ploeg

Authors
  1. T. A. Kanters
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. W. K. Redekop
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. M. E. Kruijshaar
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. A. T. van der Ploeg
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. M. P. M. H. Rutten-van Mölken
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. L. Hakkaart
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to T. A. Kanters.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Kanters, T.A., Redekop, W.K., Kruijshaar, M.E. et al. Comparison of EQ-5D and SF-6D utilities in Pompe disease. Qual Life Res 24, 837–844 (2015). https://doi.org/10.1007/s11136-014-0833-2

Download citation

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s11136-014-0833-2

Keywords

Search

Navigation