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.

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Keywords EQ-5D, Orphan disease, Pompe disease, Preference-based measure of health, SF-6D
Persistent URL dx.doi.org/10.1007/s11136-014-0833-2, hdl.handle.net/1765/81737
Journal Quality of Life Research
Kanters, T.A, Redekop, W.K, Kruijshaar, M.E, van der Ploeg, A.T, Rutten-van Mölken, M.P.M.H, & van Hakkaart-van Roijen, L. (2015). Comparison of EQ-5D and SF-6D utilities in Pompe disease. Quality of Life Research, 24(4), 837–844. doi:10.1007/s11136-014-0833-2