Arlene V. Drack
WASHINGTON — Visual benefits achieved after injection with Luxturna were not statistically significantly affected by patient age at time of treatment, according to a study presented at the American Association for Pediatric Ophthalmology and Strabismus meeting.
Arlene V. Drack, MD, reported on the 3-year results of a phase 3 trial of Luxturna (voretigene neparvovec-rzyl, Spark Therapeutics) in RPE65 mutation-associated inherited retinal disease undertaken at Children’s Hospital of Philadelphia and University of Iowa.
“For every two patients enrolled to intervention immediately, one patient was enrolled to do the exact same testing regimen for a year, but they had to wait during that year to be treated,” Drack said. “But all of these patients were treated at the end of the first year.”
At 1 year, statistically significant differences between the original intervention group and the control group in binocular mobility and in ability to see very dim light were reported previously.
The 3-year data have allowed a look at 1-year effects of treatment in all patients, in both the original intervention group and in the control group, stratified by age younger than 10 years and age 10 years and older.
“There really wasn’t a difference in treating before or after 10 years of age,” Drack said.
The youngest patient was 4 years and the oldest patient was 44 years at the time of treatment. The oldest patient achieved a two light-level of improvement, which was the typical mean of improvement. Two other adults showed no improvement at all, however.
“The take-home message is that ... there is no absolute cutoff for age, although the patients who had the least amount of improvement tended to be the oldest in the groups,” Drack said.
Furthermore, the effect was durable through 3 years of follow-up.
The treatment is FDA approved in children 1 year of age and older, so for the youngest children, pediatric ophthalmologists need to diagnose these patients with nystagmus, Drack said, “because if they have RPE65-related retinal disease, there is a treatment for them.” – by Patricia Nale, ELS
Reference: Drack AV. Year 3 results and age-stratified analyses for a phase 3 trial of voretigene neparvovec in RPE65 mutation-associated inherited retinal disease. Presented at: American Association for Pediatric Ophthalmology and Strabismus meeting; March 18-22, 2018; Washington.
Disclosure: Drack reports no relevant financial disclosures. The study has been funded by Children’s Hospital of Philadelphia and Spark Therapeutics.