Estimation of impact of RPE65-mediated inherited retinal disease on quality of life and the potential benefits of gene therapy.
Andrew J LloydNatalia PiglowskaThomas CiullaSarah PitluckScott JohnsonMarric BuessingThomas O'ConnellPublished in: The British journal of ophthalmology (2019)
This is the first study to report HRQL weights (or utilities) for health states describing different levels of vision loss in patients with IRD, 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.