Development of Novel Patient-Reported Outcome (PRO) and Observer-Reported Outcome (ObsRO) Instruments in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA): ViSIO-PRO and ViSIO-ObsRO.

Isabelle AudoFrancesco Patalano Christel NaujoksClaudio SperaM Dominik FischerJane GreenChristine KayTodd A DurhamNicola WilliamsonHelena BradleyMelissa BarclayKieran BoparaiJudit Banhazi

Published in: Ophthalmology and therapy (2023)

Results informed and supported development of the instruments to assess visual functioning symptoms and vision-dependent ADL, mobility and distal HRQoL in RP/LCA, in accordance with regulatory standards. Next steps to further support use in RP/LCA clinical trials/practice includes content and psychometric validation of the instruments in this population.

Keyphrases

patient reported outcomes
clinical trial
primary care
healthcare
anti inflammatory
transcription factor
minimally invasive
physical activity
phase ii
phase iii
placebo controlled
sleep quality