EQ-5D-5L health utility scores in Australian adults with inherited retinal diseases: A cross-sectional survey.

Publisher: Wiley-Blackwell Country of Publication: England NLM ID: 101468102 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1755-3768 (Electronic) Linking ISSN: 1755375X NLM ISO Abbreviation: Acta Ophthalmol Subsets: MEDLINE

Publication: Oxford, UK : Wiley-Blackwell
Original Publication: Oxford, UK ; Malden, MA : Blackwell Munksgaard

Quality of Life* ; Retinal Diseases*/physiopathology ; Retinal Diseases*/psychology ; Retinal Diseases*/diagnosis; Humans ; Adult ; Male ; Middle Aged ; Female ; Aged ; Cross-Sectional Studies ; Adolescent ; Young Adult ; Australia/epidemiology ; Aged, 80 and over ; Surveys and Questionnaires ; Health Status ; Visual Acuity/physiology ; Sickness Impact Profile ; Quality-Adjusted Life Years

Purpose: Economic evaluations of interventions for ocular disease require utility scores that accurately represent quality of life in the target population. This study aimed to describe the distribution of EQ-5D-5L utility values among Australian adults with symptomatic inherited retinal diseases (IRDs) and to assess the relationship between these scores and vision-related quality of life.
Methods: A survey was administered predominantly online in 2021. Participants completed the EQ-5D-5L general health utility instrument, the EQ vertical visual analogue scale (EQ-VAS) and the National Eye Institute Visual Functioning Questionnaire (NEI-VFQ-25). Self-reported IRD diagnoses were classified as being associated with central or widespread retinal involvement.
Results: Responses from 647 participants aged 18-93 years were included, 50.1% were men and 77.6% had an IRD associated with widespread retinal involvement. The majority reported no problems with self-care and no pain/discomfort but did report anxiety/depression and problems with work, study, housework, or family/leisure activities. Most people with widespread involvement reported problems with mobility. Median EQ-5D-5L utility was 0.88 and 0.91 among people with widespread and central involvement, respectively (age and sex-adjusted p = 0.029); and median EQ-VAS was 75 and 80, respectively (adjusted p = 0.003). A moderate curvilinear correlation was observed between EQ-5D-5L and NEI-VFQ-25 composite score (Spearman's ρ 0.69), but not all people with poor vision-related quality of life had low EQ-5D-5L utility values.
Conclusions: EQ-5D-5L health utility values are correlated with vision-related quality of life among adults with IRDs. However, the EQ-5D-5L may not be sensitive to the full impact of vision impairment on quality of life.
(© 2024 The Authors. Acta Ophthalmologica published by John Wiley & Sons Ltd on behalf of Acta Ophthalmologica Scandinavica Foundation.)

Austin, P.C. (2002) A comparison of methods for analyzing health‐related quality‐of‐life measures. Value in Health, 5, 329–337.
Bhadhuri, A., Dröschel, D., Guldimann, M., Jetschgo, C., Banhazi, J., Schwenkglenks, M. et al. (2022) Cost‐effectiveness of voretigene neparvovec in the treatment of patients with inherited retinal disease with RPE65 mutation in Switzerland. BMC Health Services Research, 22, 837.
Braithwaite, T., Calvert, M., Gray, A., Pesudovs, K. & Denniston, A.K. (2019) The use of patient‐reported outcome research in modern ophthalmology: impact on clinical trials and routine clinical practice. Patient Related Outcome Measures, 10, 9–24.
Britten‐Jones, A.C., Jin, R., Gocuk, S.A., Cichello, E., O'Hare, F., Hickey, D.G. et al. (2022) The safety and efficacy of gene therapy treatment for monogenic retinal and optic nerve diseases: a systematic review. Genetics in Medicine, 24, 521–534.
Britten‐Jones, A.C., O'Hare, F., Edwards, T.L., Ayton, L.N. & VENTURE Study Consortium. (2022) Victorian evolution of inherited retinal diseases natural history registry (VENTURE study): rationale, methodology and initial participant characteristics. Clinical & Experimental Ophthalmology, 50, 768–780.
Brown, M.M., Brown, G.C., Sharma, S., Kistler, J. & Brown, H. (2001) Utility values associated with blindness in an adult population. British Journal of Ophthalmology, 85, 327–331.
Browne, C., Brazier, J., Carlton, J., Alavi, Y. & Jofre‐Bonet, M. (2012) Estimating quality‐adjusted life years from patient‐reported visual functioning. Eye (London, England), 26, 1295–1301.
Clemens, S., Begum, N., Harper, C., Whitty, J.A. & Scuffham, P.A. (2014) A comparison of EQ‐5D‐3L population norms in Queensland, Australia, estimated using utility value sets from Australia, the UK and USA. Quality of Life Research, 23, 2375–2381.
Daiger, S.P., Rossiter, B.F.J., Greenberg, J., Christoffels, A. & Hide, W. (2023) RetNet, the retinal information network. Available from: https://sph.uth.edu/RetNet/ [Accessed 24th February 2023].
D'Amanda, C.S., Nolen, R., Huryn, L.A. & Turriff, A. (2020) Psychosocial impacts of Mendelian eye conditions: a systematic literature review. Survey of Ophthalmology, 65, 562–580.
De Roach, J.N., McLaren, T.L., Thompson, J.A., Hoffmann, L., Urwin, I.R., McLenachan, S. et al. (2020) The Australian inherited retinal disease registry and DNA Bank. Tasman Medical Journal, 2, 60–67.
Devlin, N.J., Shah, K.K., Feng, Y., Mulhern, B. & van Hout, B. (2018) Valuing health‐related quality of life: an EQ‐5D‐5L value set for England. Health Economics, 27, 7–22.
Farmer, C., Bullement, A., Packman, D., Long, L., Robinson, S., Nikram, E. et al. (2020) Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations: an evidence review group perspective of a NICE highly specialised technology appraisal. PharmacoEconomics, 38, 1309–1318.
Fischer, M.D., Patalano, F., Naujoks, C., Banhazi, J., Bouchet, C., O'Brien, P. et al. (2023) Psychometric validation of the ViSIO‐PRO and ViSIO‐ObsRO in retinitis pigmentosa and Leber congenital amaurosis. Ophthalmology and therapy, 12, 1359–1386.
Gandhi, M., Ang, M., Teo, K., Wong, C.W., Wei, Y.C., Tan, R.L. et al. (2020) A vision ‘bolt‐on’ increases the responsiveness of EQ‐5D: preliminary evidence from a study of cataract surgery. The European Journal of Health Economics, 21, 501–511.
Heath Jeffery, R.C., Mukhtar, S.A., McAllister, I.L., Morgan, W.H., Mackey, D.A. & Chen, F.K. (2021) Inherited retinal diseases are the most common cause of blindness in the working‐age population in Australia. Ophthalmic Genetics, 42, 431–439.
Herdman, M., Gudex, C., Lloyd, A., Janssen, M.F., Kind, P., Parkin, D. et al. (2011) Development and preliminary testing of the new five‐level version of EQ‐5D (EQ‐5D‐5L). Quality of Life Research, 20, 1727–1736.
Huygens, S.A., Versteegh, M.M., Vegter, S., Schouten, L.J. & Kanters, T.A. (2021) Methodological challenges in the economic evaluation of a gene therapy for RPE65‐mediated inherited retinal disease: the value of vision. PharmacoEconomics, 39, 383–397.
Jayasundera, K.T., Abuzaitoun, R.O., Lacy, G.D., Abalem, M.F., Saltzman, G.M., Ciulla, T.A. et al. (2022) Challenges of cost‐effectiveness analyses of novel therapeutics for inherited retinal diseases. American Journal of Ophthalmology, 235, 90–97.
Jenkinson, C., Burton, J.S., Cartwright, J., Magee, H., Hall, I., Alcock, C. et al. (2005) Patient attitudes to clinical trials: development of a questionnaire and results from asthma and cancer patients. Health Expectations, 8, 244–252.
Jiang, R., Janssen, M.F.B. & Pickard, A.S. (2021) US population norms for the EQ‐5D‐5L and comparison of norms from face‐to‐face and online samples. Quality of Life Research, 30, 803–816.
Johnson, S., Buessing, M., O'Connell, T., Pitluck, S. & Ciulla, T.A. (2019) Cost‐effectiveness of Voretigene Neparvovec‐rzyl vs standard care for RPE65‐mediated inherited retinal disease. JAMA Ophthalmology, 137, 1115–1123.
Kay, S. & Ferreira, A. (2014) Mapping the 25‐item National Eye Institute Visual Functioning Questionnaire (NEI VFQ‐25) to EQ‐5D utility scores. Ophthalmic Epidemiology, 21, 66–78.
Lacy, G.D., Abalem, M.F., Andrews, C.A., Popova, L.T., Santos, E.P., Yu, G. et al. (2021) The Michigan retinal degeneration questionnaire: a patient‐reported outcome instrument for inherited retinal degenerations. American Journal of Ophthalmology, 222, 60–68.
Lin, J.‐C. & Yu, J.‐H. (2012) Assessment of quality of life among Taiwanese patients with visual impairment. Journal of the Formosan Medical Association, 111, 572–579.
Lloyd, A., Piglowska, N., Ciulla, T., Pitluck, S., Johnson, S., Buessing, M. et al. (2019) Estimation of impact of RPE65‐mediated inherited retinal disease on quality of life and the potential benefits of gene therapy. British Journal of Ophthalmology, 103, 1610–1614.
Longworth, L., Yang, Y., Young, T., Mulhern, B., Hernández 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, 1–224.
Macedo, A.F., Hellström, A., Massof, R., Tuvesson, H., Rask, M., Ramos, P.L. et al. (2022) Predictors of problems reported on the EQ‐5D‐3L dimensions among people with impaired vision in northern Portugal. Health and Quality of Life Outcomes, 20, 132.
Mack, H.G., Britten‐Jones, A.C., McGuinness, M.B., Chen, F.K., Grigg, J.R., Jamieson, R.V. et al. (2023) Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia. Gene Therapy, 30, 336–346.
Mack, H.G., Chen, F.K., Grigg, J., Jamieson, R.V., De Roach, J., O'Hare, F. et al. (2021) Perspectives of people with inherited retinal diseases on ocular gene therapy in Australia: protocol for a National survey. BMJ Open, 11, e048361.
Malkin, A.G., Goldstein, J.E., Perlmutter, M.S. & Massof, R.W. (2013) Responsiveness of the EQ‐5D to the effects of low vision rehabilitation. Optometry and Vision Science, 90, 799–805.
Mangione, C.M. (2000) VFQ‐25 manual. Available from: https://www.rand.org/health‐care/surveys_tools/vfq.html [Accessed 24th February 2023].
Mangione, C.M., Lee, P.P., Gutierrez, P.R., Spritzer, K., Berry, S. & Hays, R.D. (2001) Development of the 25‐list‐item National Eye Institute Visual Function Questionnaire. Archives of Ophthalmology, 119, 1050–1058.
McCaffrey, N., Kaambwa, B., Currow, D.C. & Ratcliffe, J. (2016) Health‐related quality of life measured using the EQ‐5D–5L: south Australian population norms. Health and Quality of Life Outcomes, 14, 133.
McGuinness, M.B., Britten‐Jones, A.C., Ayton, L.N., Finger, R.P., Chen, F.K., Grigg, J. et al. (2022) Measurement properties of the attitudes to gene therapy for the eye (AGT‐eye) instrument for people with inherited retinal diseases. Translational Vision Science & Technology, 11, 14.
Norman, R., Mulhern, B., Lancsar, E., Lorgelly, P., Ratcliffe, J., Street, D. et al. (2023) The use of a discrete choice experiment including both duration and dead for the development of an EQ‐5D‐5L value set for Australia. PharmacoEconomics, 41, 427–438.
O'Brien, P., Enstone, A., Bridge, D., Wyn, R. & Banhazi, J. (2023) Elicitation of health state utility values in retinitis pigmentosa by time trade‐off in the United Kingdom. ClinicoEconomics and Outcomes Research, 15, 29–39.
Payakachat, N., Summers, K.H., Pleil, A.M., Murawski, M.M., Thomas, J., Jennings, K. et al. (2009) Predicting EQ‐5D utility scores from the 25‐item National Eye Institute Vision Function Questionnaire (NEI‐VFQ 25) in patients with age‐related macular degeneration. Quality of Life Research, 18, 801–813.
Peacock, S., Misajon, R., Iezzi, A., Richardson, J., Hawthorne, G. & Keeffe, J. (2008) Vision and quality of life: development of methods for the VisQoL vision‐related utility instrument. Ophthalmic Epidemiology, 15, 218–223.
Prem Senthil, M., Khadka, J., De Roach, J., Lamey, T., McLaren, T., Campbell, I. et al. (2019) Development and psychometric assessment of novel item banks for hereditary retinal diseases. Optometry and Vision Science, 96, 27–34.
Rentz, A.M., Kowalski, J.W., Walt, J.G., Hays, R.D., Brazier, J.E., Yu, R. et al. (2014) Development of a preference‐based index from the National Eye Institute Visual Function Questionnaire‐25. JAMA Ophthalmology, 132, 310–318.
Richardson, J., Iezzi, A., Khan, M.A. & Maxwell, A. (2014) Validity and reliability of the assessment of quality of life (AQoL)‐8D multi‐attribute utility instrument. Patient, 7, 85–96.
Rubin, M. (2017) Do p values lose their meaning in exploratory analyses? It depends how you define the familywise error rate. Review of General Psychology, 21, 269–275.
Sainohira, M., Yamashita, T., Terasaki, H., Sonoda, S., Miyata, K., Murakami, Y. et al. (2018) Quantitative analyses of factors related to anxiety and depression in patients with retinitis pigmentosa. PLoS ONE, 13, e0195983.
Schofield, D., Kraindler, J., Tan, O., Shrestha, R., Jelovic, D., West, S. et al. (2022) Patient‐reported health‐related quality of life in individuals with inherited retinal diseases. Ophthalmology Science, 2, 100106.
Schofield, D., Kraindler, J., Tan, O., Shrestha, R.N., West, S., Hart, N. et al. (2023) The health care and societal costs of inherited retinal diseases in Australia: a microsimulation modelling study. Medical Journal of Australia, 219, 70–76.
Shojaei, S., Sabbaghi, H., Mehrabi, Y., Daftarian, N., Etemad, K. & Ahmadieh, H. (2022) Vision‐related quality of life in patients with inherited retinal dystrophies. Journal of Current Ophthalmology, 34, 80–86.
Sugawara, T., Sato, E., Baba, T., Hagiwara, A., Tawada, A. & Yamamoto, S. (2011) Relationship between vision‐related quality of life and microperimetry‐determined macular sensitivity in patients with retinitis pigmentosa. Japanese Journal of Ophthalmology, 55, 643–646.
Tanna, P., Strauss, R.W., Fujinami, K. & Michaelides, M. (2017) Stargardt disease: clinical features, molecular genetics, animal models and therapeutic options. British Journal of Ophthalmology, 101, 25–30.
Tatour, Y. & Ben‐Yosef, T. (2020) Syndromic inherited retinal diseases: genetic, clinical and diagnostic aspects. Diagnostics, 10, 779.
Uhrmann, M.F., Lorenz, B. & Gissel, C. (2020) Cost effectiveness of voretigene neparvovec for RPE65‐mediated inherited retinal degeneration in Germany. Translational Vision Science & Technology, 9, 17.
Viriato, D., Bennett, N., Sidhu, R., Hancock, E., Lomax, H., Trueman, D. et al. (2020) An economic evaluation of voretigene neparvovec for the treatment of biallelic RPE65‐mediated inherited retinal dystrophies in the UK. Advances in Therapy, 37, 1233–1247.
Zimmermann, M., Lubinga, S.J., Banken, R., Rind, D., Cramer, G., Synnott, P.G. et al. (2019) Cost utility of voretigene neparvovec for biallelic RPE65‐mediated inherited retinal disease. Value in Health, 22, 161–167.

Retina Australia; GNT1116360 National Health and Medical Research Council; GNT1188694 National Health and Medical Research Council; GNT1195713 National Health and Medical Research Council; MRF1142962 National Health and Medical Research Council; State Government of Victoria; University of Melbourne

Keywords: health utility; inherited retinal disease; macular dystrophy; patient‐reported outcome measures; patient‐reported outcomes; retinitis pigmentosa; rod‐cone dystrophy; vision‐related quality of life

Date Created: 20240116 Date Completed: 20240708 Latest Revision: 20241021

20241021

10.1111/aos.16634

38226448