Researchers Grade Online Information Accuracy for Ocular Gene Therapeutics

Jesse D. Sengillo, MD

While gene therapies for inherited retinal diseases are fairly new, many patients are flocking to online searches to try to learn as much as they can about these potential therapeutics.

A team, led by Jesse D. Sengillo, MD, Bascom Palmer Eye Institute, University of Miami, the American Society of Retina Specialists 2020 (ASRS 2020) Virtual Sessions on the accuracy and quality of information available to patients online for ocular gene therapy.

The advent of gene therapy for RPE-65-associated Leber Congenital Amaurosis has caused many to turn to internet searches for information on treatment for inherited retinal disease. While the available information is often nuanced and technical, it can require the guidance of a specialist or genetic counselor.

In the cross-sectional analysis of online resources describing ocular gene therapy, the investigators identified resources, entered keywords “eye gene therapy” or “Luxturna” into Google search engines and medical websites.

Ultimately, they chose websites from the American Academy of Ophthalmology, American Optometric Association, EyeWiki, U.S. Food and Drug Administration (FDA), Spark Therapeutics, MedicineNet, National Eye Institute, Novartis, WebMD, and Wikipedia.

They then created a 25-question assessment and applied using 3 graders to each article.

The researchers quantified inter-observe reproducibility with Pearson’s correlation and evaluated article accountability using JAMA benchmarks.

The average questionnaire score for all of the articles was 33.93 out of 100 possible points.

The investigators found a statistically significant difference in the content accuracy and quality between different articles (P = 0.017). EyeWiki achieved the highest score at 51.33 and MedicineNet had the lowest with 16.67. Inter-grader reproducibility was statistically significant between grader 1 and both grader 2 and 3 (r = 0.66; P = 0.038; r = -.84; P = 0.003, respectively).

The difference in content accuracy and quality also approached statistically significant between 2 and 4 (P = 0.058).

None of the articles achieved all 4 JAMA benchmarks, with only 1 of the 10 articles achieving 3 JAMA benchmarks. Currency was the most displayed benchmark, followed by attribution and authorship.

“There is paucity of online resources available to patients with questions regarding ocular gene therapy,” the authors wrote. “Information online is not of sufficient quality to aid patients in understanding current treatments. With the approval of Vortigene neparvovec and multiple other ocular gene-based therapies in the pipeline, there is a need for improved or new resources to ensure patients are well-informed.”

Luxturna was the first therapy to inherited retinal disease after it was approved by the FDA in 2017. In 2019, researchers presented data that children have been successfully treated with for inherited vision loss.

Investigators from the Kellogg Eye Center explored gene therapy with Luxturna in order to treat adult and pediatric patients with inherited forms of vision loss. Various retinal dystrophies can lead to progressive vision loss or total blindness, including retinitis pigmentosa, Stargardt disease, Leber congenital amaurosis, achromatopsia, X-linked retinoschisis, Usher syndrome, and choroideremia.

The study, “Assessing the Accuracy and Quality of Patient Accessible Online Resources for Ocular Gene Therapy,” was published online by ASRS 2020.