Healio
Healio
June 19, 2017
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Decreased autofluorescence potential outcome measure for Stargardt disease trials

In patients with Stargardt disease, 50% of eyes without definitely decreased autofluorescence at first visit may develop atrophic lesions of the retinal pigment epithelium in less than 5 years, which could prove to be a clinical outcome measure for treatment trials in the future, according to a study.

The retrospective multicenter cohort study included 390 eyes of 217 patients who were age 6 years and older and had Stargardt disease and pathogenic mutations in the ABCA4 gene. All patients underwent fundus autofluorescence imaging for at least two visits with a minimum of 6 months between at least two visits.

At first visit, 14.6% of the eyes did not exhibit any flecks, while 52.8% of eyes at first visit did not have any area of definitely decreased autofluorescence (DDAF). Researchers found that the median time to develop DDAF lesions in these patients was 4.9 years. By 3 years, 24% of patients had developed lesions.

In patients without questionably decreased autofluorescence (QDAF) lesions, the median time to develop QDAF lesions was 6.3 years.

“Because the median time for the development of DDAF lesions was 4.93 years in our cohort, this may be a suitable outcome measure in studies with longer follow-up. Similarly, the incidence of QDAF lesions provided further insight into the progression of Stargardt disease,” the researchers wrote. – by Robert Linnehan

Disclosures: Strauss reports receiving support from the Austrian Science Fund and Foundation Fighting Blindness Clinical Research Institute. Please see the study for all other authors’ relevant financial disclosures.

Sources/Disclosures

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Source:

Strauss RW, et al. JAMA Ophthalmol. 2017;doi:10.1001/jamaophthalmol.2017.1121.