Ranked Importance of Visual Function Outcome Measures in Choroideremia Clinical Trials.

ABSTRACT:

Purpose

Clinical trials of novel therapies for choroideremia require robust and clinically meaningful visual function outcome measures. Best-corrected visual acuity (BCVA) is mostly insensitive to changes in disease state, until late stages, and hence also to potential therapeutic gains after gene therapies. While the insensitivity of BCVA as an effective outcome measure is common wisdom, its low importance has not been rigorously demonstrated in the literature. This work uses statistical techniques to rank the relative importance of potential functional outcome measures in choroideremia.

Methods

Retrospective dominance analysis was performed on data from a longitudinal interventional clinical trial performed at the Oxford Eye Hospital. Functional data from the untreated eye

SUBMITTER: Josan AS 

PROVIDER: S-EPMC11605660 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature