July 2019
Volume 60, Issue 9
Open Access
ARVO Annual Meeting Abstract  |   July 2019
Estimation of Impact of RPE65-Mediated Inherited Retinal Disease on Quality of Life
Author Affiliations & Notes
  • Thomas A Ciulla
    Retina, Midwest Eye Institute, Indianapolis, Indiana, United States
    Spark Therapeutics (previously); Clearside Biomedical, Alpharetta, Georgia, United States
  • Andrew Lloyd
    Acaster Lloyd Consulting Ltd, London, United Kingdom
  • Natalia Piglowska
    Acaster Lloyd Consulting Ltd, London, United Kingdom
  • Sarah Pitluck
    Spark Therapeutics, Philadelphia, Pennsylvania, United States
  • Scott Johnson
    Medicus Economics, Boston, Massachusetts, United States
  • Marric Buessing
    Medicus Economics, Boston, Massachusetts, United States
  • Thomas O'Connell
    Medicus Economics, Boston, Massachusetts, United States
  • Footnotes
    Commercial Relationships   Thomas Ciulla, Clearside Biomedical (E), Clearside Biomedical (I), Spark Therapeutics (E); Andrew Lloyd, None; Natalia Piglowska, None; Sarah Pitluck, Spark Therapeutics (E), Spark Therapeutics (I); Scott Johnson, None; Marric Buessing, None; Thomas O'Connell, None
  • Footnotes
    Support  Spark Therapeutics
Investigative Ophthalmology & Visual Science July 2019, Vol.60, 4466. doi:
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      Thomas A Ciulla, Andrew Lloyd, Natalia Piglowska, Sarah Pitluck, Scott Johnson, Marric Buessing, Thomas O'Connell; Estimation of Impact of RPE65-Mediated Inherited Retinal Disease on Quality of Life. Invest. Ophthalmol. Vis. Sci. 2019;60(9):4466.

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

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Abstract

Purpose : As the health care system evolves from a volume-based system to a value-based system, health-related quality of life (HRQL) data becomes increasingly important, but in rare diseases can be difficult to capture. Given the ultra-rare nature of RPE65-mediated inherited retinal disease (IRD), it was not feasible to recruit a patient sample and collect HRQL data prospectively. This study estimated quality of life weights (utilities) related to different levels of vision loss in RPE65-mediated IRD.

Methods : Vignette descriptions of IRD states were developed and then assessed to elicit utilities. The vignettes ranged from moderate vision loss through hand motion to no light perception (NLP). Six retina specialists with additional expertise in IRDs provided a proxy valuation of the vignettes using generic measures of health – the EQ-5D-5L and Health Utility Index 3 (HUI3). The data were then scored using standard methods for each instrument.

Results : Weights from both HRQL measures revealed a large decline in scores with vision loss. The EQ-5D-5L weights ranged from 0.709 for moderate vision loss to 0.152 for hand motion to NLP. The HUI3 weights ranged from 0.519 to -0.039, respectively. A substantial decline was seen on both measures, and the degree of decline from moderate vision loss to NLP was identical on both (-0.56).

Conclusions : This is the first study to report HRQL weights (or utilities) for health states describing different levels of vision loss in IRD patients, specifically those with RPE65-mediated disease. The parallel decline in scores from the EQ-5D and HUI3 corroborates the substantial impact of progressive vision loss on HRQL.

This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.

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