June 2022
Volume 63, Issue 7
Open Access
ARVO Annual Meeting Abstract  |   June 2022
Psychometric validation of patient-reported outcome (PRO) and observer-reported outcome (ObsRO) measures in Retinitis Pigmentosa and Leber Congenital Amaurosis
Author Affiliations & Notes
  • Isabelle S Audo
    Sorbonne Université, INSERM, CNRS, Institut de la Vision, CHNO des Quinze-Vingts, REFERET national rare disease center, Paris, France
  • Nicola Williamson
    Adelphi Mill, Bollington, Cheshire, United Kingdom
  • Melissa Barclay
    Adelphi Mill, Bollington, Cheshire, United Kingdom
  • Joel Sims
    Adelphi Mill, Bollington, Cheshire, United Kingdom
  • Kieran Boparai
    Adelphi Mill, Bollington, Cheshire, United Kingdom
  • Francesco Patalano
    Novartis Pharma AG, Basel, Basel-Stadt, Switzerland
  • Christel Naujoks
    Novartis Pharma AG, Basel, Basel-Stadt, Switzerland
  • Claudio Spera
    Novartis Pharma AG, Basel, Basel-Stadt, Switzerland
  • Judit Banhazi
    Novartis Pharma AG, Basel, Basel-Stadt, Switzerland
  • Paul O’Brien
    Novartis Ireland Ltd, Dublin, Ireland
  • Christine Kay
    University of Florida, Gainesville, Florida, United States
  • Jane Green
    Discipline of Genetics, Faculty of Medicine, Memorial University of Newfoundland, St. John's, Newfoundland, Canada
  • Todd Durham
    Foundation Fighting Blindness Inc, Columbia, Maryland, United States
  • Helena Bradley
    Adelphi Mill, Bollington, Cheshire, United Kingdom
  • M Dominik Fischer
    Centre for Ophthalmology, University of Tübingen, Tübingen, Germany
    Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, United Kingdom
  • Footnotes
    Commercial Relationships   Isabelle Audo Adelphi Values, Novartis, Sparing Vision, Roche, and Biogen, Code C (Consultant/Contractor); Nicola Williamson Adelphi Values, Code E (Employment); Melissa Barclay Adelphi Values, Code E (Employment); Joel Sims Adelphi Values, Code E (Employment); Kieran Boparai Adelphi Values, Code E (Employment); Francesco Patalano Novartis Pharma AG, Code E (Employment); Christel Naujoks Novartis Pharma AG, Code E (Employment); Claudio Spera Novartis Pharma AG, Code E (Employment); Judit Banhazi Novartis Pharma AG, Code E (Employment); Paul O’Brien Novartis Ireland Ltd, Dublin, Ireland, Code E (Employment); Christine Kay AGTC, Foundation Fighting Blindness, Alkeus, Gyroscope, REGENXBIO, NightstarTherapeutics/Biogen, Spark therapeutics, Novartis, Iveric Bio, ProQR Therapeutics, MeiraGTx, Janssen,Atsena Therapeutics, 4D Molecular Therapeutics, and Kodiak, Code C (Consultant/Contractor); Jane Green Adelphi Values, Code C (Consultant/Contractor); Todd Durham Novartis, Code C (Consultant/Contractor); Helena Bradley Adelphi Values, Code E (Employment); M Dominik Fischer Adelphi Values, Advent France Biotechnology, Alphasights, Atheneum, Axiom Healthcare Strategies, Biogen, Decision Resources, Dialectica, Frontera Therapeutics, Janssen Research & Development, Navigant, Novartis, Roche, Sirion, and STZ eyetrial, Code C (Consultant/Contractor)
  • Footnotes
    Support  Novartis Pharma AG
Investigative Ophthalmology & Visual Science June 2022, Vol.63, 2663. doi:
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      Isabelle S Audo, Nicola Williamson, Melissa Barclay, Joel Sims, Kieran Boparai, Francesco Patalano, Christel Naujoks, Claudio Spera, Judit Banhazi, Paul O’Brien, Christine Kay, Jane Green, Todd Durham, Helena Bradley, M Dominik Fischer; Psychometric validation of patient-reported outcome (PRO) and observer-reported outcome (ObsRO) measures in Retinitis Pigmentosa and Leber Congenital Amaurosis. Invest. Ophthalmol. Vis. Sci. 2022;63(7):2663.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) are rare inherited retinal degenerative disorders. There is no RP/LCA-specific measure of visual impairments and impacts on vision-dependent activities of daily living (ADL). The Visual Symptom and Impact Outcomes patient-reported outcome (ViSIO-PRO) and observer-reported outcome (ViSIO-ObsRO) measures were developed through qualitative research with RP/LCA patients, caregivers of child patients and expert clinicians. This study evaluated the psychometric properties of the instruments.

Methods : The 49-item ViSIO-PRO and 27-item ViSIO-ObsRO instruments were completed by 83 adult and adolescent patients and 22 caregivers of child patients aged 3-11 years with RP/LCA, respectively, at baseline and 12-16-day follow-up. Concurrent measures were also administered at BL. Psychometric analyses assessed item (question) properties, dimensionality, scoring, reliability, validity, and score interpretation.

Results : Responses to the ViSIO-PRO and ViSIO-ObsRO items were mainly evenly distributed across response options. Inter-item correlations were mostly moderate-to-strong (>0.30) at baseline within hypothesized domains. Item properties, qualitative data and clinical input supported retention of 35 ViSIO-PRO items and 25 ViSIO-ObsRO items. Confirmatory factor analysis supported a four-factor model (visual function symptoms, mobility, vision-dependent ADL and broader health-related quality of life). A bi-factor model supported calculation of total scores and domain scores. Internal consistency was high for all domain and total scores (Cronbach’s alpha>0.70). Test-retest reliability for the total scores was strong between BL and 12-16-day follow-up (Intraclass Correlation Coefficients=0.66-0.98). Convergent validity was supported by strong correlations in a logical pattern with concurrent measures at baseline. There were statistically significant differences in mean ViSIO-PRO and ViSIO-ObsRO scores between known-groups defined by global impression items at baseline. Distribution-based methods provide initial insights to guide interpretation of scores.

Conclusions : Findings supported item reduction, established the scoring of the ViSIO-PRO and ViSIO-ObsRO instruments, and provided evidence of reliability and validity as outcome measures in RP/LCA.

This abstract was presented at the 2022 ARVO Annual Meeting, held in Denver, CO, May 1-4, 2022, and virtually.

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