caa a22 4500 606179682 CHVBK 20210128100802.0 cr unu---uuuuu 210128e20150401xx s 000 0 eng 10.1007/s11136-014-0833-2 doi (NATIONALLICENCE)springer-10.1007/s11136-014-0833-2 Comparison of EQ-5D and SF-6D utilities in Pompe disease [Elektronische Daten] [T. Kanters, W. Redekop, M. Kruijshaar, A. van der Ploeg, M. Rutten-van Mölken, L. Hakkaart] 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. Springer International Publishing Switzerland, 2014 Preference-based measure of health nationallicence EQ-5D nationallicence SF-6D nationallicence Orphan disease, Pompe disease nationallicence Kanters T. Department of Health Policy and Management, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Burgemeester Oudlaan 50, P.O. Box 1738, 3000, Rotterdam, DR, The Netherlands aut Redekop W. Department of Health Policy and Management, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Burgemeester Oudlaan 50, P.O. Box 1738, 3000, Rotterdam, DR, The Netherlands aut Kruijshaar M. Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Dr Molewaterplein 60, 3015, Rotterdam, GJ, The Netherlands aut van der Ploeg A. Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Dr Molewaterplein 60, 3015, Rotterdam, GJ, The Netherlands aut Rutten-van Mölken M. Department of Health Policy and Management, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Burgemeester Oudlaan 50, P.O. Box 1738, 3000, Rotterdam, DR, The Netherlands aut Hakkaart L. Department of Health Policy and Management, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Burgemeester Oudlaan 50, P.O. Box 1738, 3000, Rotterdam, DR, The Netherlands aut Quality of Life Research Springer International Publishing 24/4(2015-04-01), 837-844 0962-9343 24:4<837 2015 24 11136 https://doi.org/10.1007/s11136-014-0833-2 text/html Onlinezugriff via DOI BK010053 XK010053 XK010000 Metadata rights reserved Springer special CC-BY-NC licence nationallicence 1 research-article jats NATIONALLICENCE NATIONALLICENCE NL-springer NATIONALLICENCE 856 40 https://doi.org/10.1007/s11136-014-0833-2 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Kanters T. Department of Health Policy and Management, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Burgemeester Oudlaan 50, P.O. Box 1738, 3000, Rotterdam, DR, The Netherlands aut NATIONALLICENCE 700 1- Redekop W. Department of Health Policy and Management, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Burgemeester Oudlaan 50, P.O. Box 1738, 3000, Rotterdam, DR, The Netherlands aut NATIONALLICENCE 700 1- Kruijshaar M. Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Dr Molewaterplein 60, 3015, Rotterdam, GJ, The Netherlands aut NATIONALLICENCE 700 1- van der Ploeg A. Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Dr Molewaterplein 60, 3015, Rotterdam, GJ, The Netherlands aut NATIONALLICENCE 700 1- Rutten-van Mölken M. Department of Health Policy and Management, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Burgemeester Oudlaan 50, P.O. Box 1738, 3000, Rotterdam, DR, The Netherlands aut NATIONALLICENCE 700 1- Hakkaart L. Department of Health Policy and Management, Institute for Medical Technology Assessment, Erasmus University Rotterdam, Burgemeester Oudlaan 50, P.O. Box 1738, 3000, Rotterdam, DR, The Netherlands aut NATIONALLICENCE 773 0- Quality of Life Research Springer International Publishing 24/4(2015-04-01), 837-844 0962-9343 24:4<837 2015 24 11136