Quality of Life Research

, Volume 24, Issue 4, pp 837–844 | Cite as

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

  • T. A. Kanters
  • W. K. Redekop
  • M. E. Kruijshaar
  • A. T. van der Ploeg
  • M. P. M. H. Rutten-van Mölken
  • L. Hakkaart



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.


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.


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.


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.


Preference-based measure of health EQ-5D SF-6D Orphan disease, Pompe disease 



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.


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Copyright information

© Springer International Publishing Switzerland 2014

Authors and Affiliations

  • T. A. Kanters
    • 1
    • 2
  • W. K. Redekop
    • 1
  • M. E. Kruijshaar
    • 2
    • 3
  • A. T. van der Ploeg
    • 2
    • 3
  • M. P. M. H. Rutten-van Mölken
    • 1
  • L. Hakkaart
    • 1
  1. 1.Department of Health Policy and Management, Institute for Medical Technology AssessmentErasmus University RotterdamRotterdamThe Netherlands
  2. 2.Center for Lysosomal and Metabolic DiseasesErasmus MC University Medical CenterRotterdamThe Netherlands
  3. 3.Division of Metabolic Diseases and Genetics, Department of PediatricsErasmus MC University Medical Center RotterdamRotterdamThe Netherlands

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