A review of preference-based measures for the assessment of quality of life in children and adolescents with cerebral palsy
To examine the psychometric properties and suitability for use within the context of cerebral palsy research in children and adolescents of generic preference-based outcome measures (PROMs).
Nine electronic databases were searched in this systematic review. The consensus-based standards for the selection of health measurement instruments (COSMIN) checklist were used to measure the psychometric properties of the PROMs. A meta-analysis was used to pool correlation coefficients for convergent validity using the Schmidt–Hunter method. Heterogeneity was assessed using the I-squared statistic (I2).
Four preference-based PROMs were identified from eight studies: Health Utilities Index—Mark 2 and 3 (HUI-2 and HUI-3, respectively), the Assessment Quality of Life-4 dimension (AQoL-4D) and the EuroQol-5 dimension 3 level (EQ-5D-3L). Only the HUI system was primarily developed for application with children/adolescents though health-state values for scoring the PROM were elicited from adults. The HUI-3 covered the most relevant constructs though it excludes important modules of health-related quality of life (HRQOL) such as activity limitations and participation restrictions. In terms of psychometric properties, evidence was presented for only five of COSMIN measurement properties: reliability (HUI3), measurement error (HUI-3), content validity (HUI-2 and HUI-3), Hypotheses testing (HUI-3 and AQoL-4D) and criterion validity (HUI-3). No papers reported on internal consistency, structural validity, cross-cultural validity or responsiveness of the preference-based measures in children and adolescents with cerebral palsy.
This review highlights the dearth in studies using preference-based PROMs to measure HRQOL associated with cerebral palsy in children and adolescents. The HUI-3 demonstrated the strongest psychometric properties, though it does not cover all dimensions relevant to this population.
KeywordsQuality of life Utility Instruments Child Adolescent Cerebral palsy
The Sixteen-dimensional measure of HRQOL
The adolescent health utility measure
Assessment Quality of Life
Child health utility 9-dimensions
Cerebral palsy quality of life
EuroQol-5 dimensions 3 levels
EuroQol-5 dimensions youth version
Gross motor function classification system
Health-related quality of life
Health Utilities Index
National Institute for Health and Care Excellence
Pharmaceutical Benefits Advisory Committee
Pediatric Quality of Life Inventory
Preferred reporting items for systematic reviews and meta-analysis
Patient-reported outcome measures
International Prospective Register of Systematic Reviews
Quality of life
Quality of life instrument for people with developmental disability
Quality of well-being scale
Visual analogue scale
CM is supported by the Australian Government Research Training Program Scholarship.
CM, GC and JR formulated the idea for the study. CM wrote the first draft and the co-authors (EH, GC, RR, JR) revised the study for important intellectual content. CM will act as a guarantor for the work.
Compliance with ethical standards
Conflict of interest
All authors declared no conflict of interest.
Research involving human and animal rights
This manuscript is a systematic review which only contains data from previously published studies. No clinical trials were conducted nor were patient data collected for this research.
- 1.National Institute for Health and Care Excellence NICE. (2013). Guide to the methods of technology appraisal 2013.Google Scholar
- 3.Acquadro, C., Berzon, R., Dubois, D., Leidy, N. K., Marquis, P., Revicki, D., et al. (2003). Incorporating the patient’s perspective into drug development and communication: An ad hoc task force report of the patient-reported outcomes (PRO) harmonization group meeting at the Food and Drug Administration, February 16, 2001. Value Health, 6(5), 522–531. https://doi.org/10.1046/j.1524-4733.2003.65309.x.CrossRefPubMedGoogle Scholar
- 5.Brazier, J., Ratcliffe, J., Salmon, J., A., & Tsuchiya, A. (2017). Measuring and valuing health benefits for economic evaluation. London: Oxford University Press.Google Scholar
- 7.Drummond, M. F., Torrance, G. W., O’Brien, B. J., & Stoddart, G. L. (2005). Methods for the economic evaluation of health care programmes. Oxford: Oxford University Press.Google Scholar
- 8.Neumann, P. J., Goldie, S. J., & Weinstein, M. C. (2000). Preference-based measures in economic evaluation in health care. Annual Review of Public Health, 21(1), 587–611. https://doi.org/10.1146/annurev.publhealth.21.1.587.CrossRefPubMedGoogle Scholar
- 10.Harris, A. B. L. (2004). Getting value for money: “The Australian experience”. In M. –H. International (Ed.), Health care coverage determinations: An international comparative study. Maidenhead: Open University Press.Google Scholar
- 11.National Institute for Health and Care Excellence. (2012). Appendix G: Methodology checklist: Economic evaluations (PMG6B).Google Scholar
- 14.Davis, E., Waters, E., Mackinnon, A., Reddihough, D., Graham, H. K., Mehmet-Radji, O., et al. (2006). Paediatric quality of life instruments: A review of the impact of the conceptual framework on outcomes. Developmental Medicine & Child Neurology, 48(4), 311–318. https://doi.org/10.1017/s0012162206000673.CrossRefGoogle Scholar
- 15.Carlon, S., Shields, N., Yong, K., Gilmore, R., Sakzewski, L., & Boyd, R. (2010). A systematic review of the psychometric properties of quality of life measures for school aged children with cerebral palsy. BMC Pediatrics, 10, 81. https://doi.org/10.1186/1471-2431-10-81.CrossRefPubMedPubMedCentralGoogle Scholar
- 16.Janssens, A., Rogers, M., Gumm, R., Jenkinson, C., Tennant, A., Logan, S., et al. (2016). Measurement properties of multidimensional patient-reported outcome measures in neurodisability: A systematic review of evaluation studies. Developmental Medicine & Child Neurology, 58(5), 437–451. https://doi.org/10.1111/dmcn.12982.CrossRefGoogle Scholar
- 18.Morris, C., Kurinczuk, J. J., & Fitzpatrick, R. (2005). Child or family assessed measures of activity performance and participation for children with cerebral palsy: A structured review. Child: Care, Health and Development, 31(4), 397–407. https://doi.org/10.1111/j.1365-2214.2005.00519.x.Google Scholar
- 19.Mokkink, L. B., Terwee, C. B., Patrick, D. L., Alonso, J., Stratford, P. W., Knol, D. L., et al. (2010). The COSMIN checklist for assessing the methodological quality of studies on measurement properties of health status measurement instruments: An international Delphi study. Quality of Life Research, 19(4), 539–549. https://doi.org/10.1007/s11136-010-9606-8.CrossRefPubMedPubMedCentralGoogle Scholar
- 20.Mulhern, B., Mukuria, C., Barkham, M., Knapp, M., Byford, S., Soeteman, D., et al. (2014). Using generic preference-based measures in mental health: Psychometric validity of the EQ-5D and SF-6D. British Journal of Psychiatry, 205(3), 236–243. https://doi.org/10.1192/bjp.bp.112.122283.CrossRefPubMedGoogle Scholar
- 23.Longworth, L., Yang, Y., Young, T., Mulhern, B., Hernandez Alava, M., Mukuria, C., et al. (2014). Use of generic and condition-specific measures of health-related quality of life in NICE decision-making: A systematic review, statistical modelling and survey. Health Technology Assessment, 18(9), 1–224. https://doi.org/10.3310/hta18090.CrossRefPubMedPubMedCentralGoogle Scholar
- 24.Brazier, J. E., Rowen, D., Mavranezouli, I., Tsuchiya, A., Young, T., Yang, Y., et al. (2012). Developing and testing methods for deriving preference-based measures of health from condition-specific measures (and other patient-based measures of outcome). Health Technology Assessment, 16(32), 1–114. https://doi.org/10.3310/hta16320.CrossRefGoogle Scholar
- 27.Findlay, B., Switzer, L., Narayanan, U., Chen, S., & Fehlings, D. (2016). Investigating the impact of pain, age, gross motor function classification system, and sex on health-related quality of life in children with cerebral palsy. Developmental Medicine & Child Neurology, 58(3), 292–297. https://doi.org/10.1111/dmcn.12936.CrossRefGoogle Scholar
- 28.Chen, K. L., Tseng, M. H., Shieh, J. Y., Lu, L., & Huang, C. Y. (2014). Determinants of quality of life in children with cerebral palsy: A comprehensive biopsychosocial approach. Research in Developmental Disabilities, 35(2), 520–528. https://doi.org/10.1016/j.ridd.2013.12.002.CrossRefPubMedGoogle Scholar
- 30.Narayanan, U. G., Fehlings, D., Weir, S., Knights, S., Kiran, S., & Campbell, K. (2006). Initial development and validation of the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD). Developmental Medicine & Child Neurology, 48(10), 804–812. https://doi.org/10.1017/s0012162206001745.CrossRefGoogle Scholar
- 33.Mpundu-Kaambwa, C., Chen, G., Huynh, E., Russo, R., & Ratcliffe, J. (2017). Protocol for a systematic review of instruments for the assessment of quality of life and well-being in children and adolescents with cerebral palsy. British Medical Journal Open, 7(9), e015924. https://doi.org/10.1136/bmjopen-2017-015924.Google Scholar
- 34.Liberati, A., Altman, D. G., Tetzlaff, J., Mulrow, C., Gotzsche, P. C., Ioannidis, J. P., et al. (2009). The PRISMA statement for reporting systematic reviews and meta-analyses of studies that evaluate health care interventions: Explanation and elaboration. PLoS Medicine. 6. https://doi.org/10.1371/journal.pmed.1000100.Google Scholar
- 35.Terwee, C. B., Mokkink, L. B., Knol, D. L., Ostelo, R. W., Bouter, L. M., & de Vet, H. C. (2012). Rating the methodological quality in systematic reviews of studies on measurement properties: A scoring system for the COSMIN checklist. Quality of Life Research, 21(4), 651–657. https://doi.org/10.1007/s11136-011-9960-1.CrossRefPubMedGoogle Scholar
- 39.Hawthorne, G., Richardson, J., & Day, N. A. (2001). A comparison of the Assessment of Quality of Life (AQoL) with four other generic utility instruments. Annals of Internal Medicine, 33(5), 358–370.Google Scholar
- 42.Livingston, M. H., & Rosenbaum, P. L. (2008). Adolescents with cerebral palsy: Stability in measurement of quality of life and health-related quality of life over 1 year. Developmental Medicine & Child Neurology, 50(9), 696–701. https://doi.org/10.1111/j.1469-8749.2008.03053.x.CrossRefGoogle Scholar
- 43.Rosenbaum, P., Livingston, M. H., Palisano, R. J., Galuppi, B. E., & Russell, D. J. (2007). Quality of life and health-related quality of life of adolescents with cerebral palsy. Developmental Medicine & Child Neurology, 49(7), 516–521. https://doi.org/10.1111/j.1469-8749.2007.00516.x.CrossRefGoogle Scholar
- 47.Young, N. L., Rochon, T. G., McCormick, A., Law, M., Wedge, J. H., & Fehlings, D. (2010). The health and quality of life outcomes among youth and young adults with cerebral palsy. The Archives of Physical Medicine and Rehabilitation, 91(1), 143–148. https://doi.org/10.1016/j.apmr.2009.08.152.CrossRefPubMedGoogle Scholar
- 48.Hoving, M. A., Evers, S. M. A. A., Ament, A. J. H. A., Van Raak, E. P. M., & Vles, J. S. H. (2008). Intrathecal baclofen therapy in children with intractable spastic cerebral palsy: A cost-effectiveness analysis. Developmental Medicine & Child Neurology, 50(6), 450–455. https://doi.org/10.1111/j.1469-8749.2008.02059.x.CrossRefGoogle Scholar
- 57.Feeny, D., Furlong, W., Barr, R. D., Torrance, G. W., Rosenbaum, P., & Weitzman, S. (1992). A comprehensive multiattribute system for classifying the health status of survivors of childhood cancer. Journal of Clinical Oncology, 10(6), 923–928. https://doi.org/10.1200/jco.19184.108.40.2063.CrossRefPubMedGoogle Scholar
- 58.Feeny, D. H., Torrance, G. W., & Furlong, W. J. (1996). Health Utilities Index. In B. Spiker (Ed.), Quality of life and pharmacoeconomics in clinical trials ((2nd ed., pp. 239–252). Philadelphia: Linppincott-Raven.Google Scholar
- 61.StatsDirect Ltd. (2013). StatsDirect statistical software. Retrieved August 21, 2017, from http://www.statsdirect.com.
- 62.Moodie, M., Richardson, J., Rankin, B., Iezzi, A., & Sinha, K. (2010). Predicting time trade-off health state valuations of adolescents in four Pacific countries using the Assessment of Quality-of-Life (AQoL-6D) instrument. Value Health, 13(8), 1014–1027. https://doi.org/10.1111/j.1524-4733.2010.00780.x.CrossRefPubMedGoogle Scholar
- 70.Raphael, D., Brown, I., Renwick, R., & Rootman, I. (1996). Assessing the quality of life of persons with developmental disabilities: Description of a new model, measuring instruments, and initial findings. International Journal of Disability, Development and Education, 43(1), 25–42. https://doi.org/10.1080/0156655960430103.CrossRefGoogle Scholar
- 79.Ratcliffe, J., Stevens, K., Flynn, T., Brazier, J., & Sawyer, M. G. (2012). Whose values in health? An empirical comparison of the application of adolescent and adult values for the CHU-9D and AQOL-6D in the Australian adolescent general population. Value in Health, 15(5), 730–736. https://doi.org/10.1016/j.jval.2012.04.005.CrossRefPubMedGoogle Scholar
- 80.Ratcliffe, J., Couzner, L., Flynn, T., Sawyer, M., Stevens, K., Brazier, J., et al. (2011). Valuing child health utility 9D health states with a young adolescent sample. Applied Health Economics and Health Policy, 9(1), 15–27. https://doi.org/10.2165/11536960-000000000-00000.CrossRefPubMedGoogle Scholar
- 82.Stevens, K., & Ratcliffe, J. (2012). Measuring and valuing health benefits for economic evaluation in adolescence: An assessment of the practicality and validity of the child health utility 9D in the Australian adolescent population. Value Health, 15(8), 1092–1099. https://doi.org/10.1016/j.jval.2012.07.011.CrossRefPubMedGoogle Scholar
- 84.White-Koning, M., Arnaud, C., Dickinson, H. O., Thyen, U., Beckung, E., Fauconnier, J., et al. (2007). Determinants of child-parent agreement in quality-of-life reports: A European study of children with cerebral palsy. Pediatrics, 120(4), e804–814. https://doi.org/10.1542/peds.2006-3272.CrossRefPubMedGoogle Scholar
- 86.National Health and Hospitals Reform Commission (2010). A healthier future for all Australians—Final report June 2009.Google Scholar