Assessment of visual function in patients with gyrate atrophy who are considered candidates for gene replacement.

Abstract

OBJECTIVE:

To assess the course of change of visual function outcome variables in 5 patients with gyrate atrophy before a gene replacement therapy clinical trial.

METHODS:

The outcome variables selected were visual field sensitivity and electroretinogram amplitude. The course of change of these outcome variables was determined by calculation of their half-lives.

RESULTS:

In the 4 to 6 years during which each patient was followed up for this study, median visual field half-lives were 17.0 years (static perimetry) and 11.4 years (kinetic perimetry). Median electroretinogram half-lives were 16.0 years (maximal response) and 10.7 years (flicker response).

CONCLUSIONS:

The course of the decline of visual function outcome variables is frequently slow. Thus, a long-term clinical trial would be required to assess the efficacy of the intervention in the preservation of visual function.

• Authors: R C Caruso, R B Nussenblatt, K G Csaky, D Valle, M I Kaiser-Kupfer
• Journal: Archives of ophthalmology (Chicago, Ill. : 1960) (Arch Ophthalmol) Vol. 119 Issue 5 Pg. 667-9 (May 2001) ISSN: 0003-9950 [Print] United States
• PMID: 11346393 (Publication Type: Journal Article)

Topics

• Adult
• Aged
• Electroretinography
• Genetic Therapy
• Gyrate Atrophy (physiopathology, therapy)
• Humans
• Middle Aged
• Retina (physiopathology)
• Visual Acuity (physiology)
• Visual Field Tests
• Visual Fields (physiology)