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Rafael C. Caruso, MD; Robert B. Nussenblatt, MD; Karl G. Csaky, MD, PhD; David Valle, MD; Muriel I. Kaiser-Kupfer, MD

Arch Ophthalmol. 2001;119:667-669.

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.

From the Ophthalmic Genetics and Visual Function Branch (Drs Caruso and Kaiser-Kupfer) and Laboratory of Immunology (Drs Nussenblatt and Csaky), National Eye Institute, National Institutes of Health, Bethesda, Md; and Howard Hughes Medical Institute Research Laboratories, The Johns Hopkins University School of Medicine, Baltimore, Md (Dr Valle).

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