Drug therapy shows promise in treating leading cause of childhood blindness

LONDON - The results of a multicenter clinical trial, led by researchers at The University of Texas Health Science Center at Houston (UTHealth), hold potentially good news for preterm infants who develop an eye condition that may cause blindness.

A readily available, inexpensive drug therapy showed a significant benefit in treating premature infants with the worst and historically most difficult-to-treat cases of retinopathy of prematurity.

Retinopathy of prematurity is a leading cause of childhood blindness worldwide. In the immature retina of babies born before 30 weeks’ gestational age, the disease results in disorganized growth of retinal blood vessels, which can lead to scarring and retinal detachment.

In this study, Helen A. Mintz-Hittner, the Alfred W. Lasher, III, Professor in the Department of Ophthalmology and Visual Science at the UTHealth Medical School, and colleagues compared the use of intravitreal bevacizumab, an anti-vascular endothelial growth factor, to conventional laser treatment.

The study investigators treated infants with acute retinopathy of prematurity affecting zone I and posterior zone II - the retinal zones with the highest rate of treatment failure.

Data on the outcomes of 143 infants enrolled in the study showed that, among infants with zone I disease, the recurrence rate was 6 percent with intravitreal bevacizumab and 42 percent with conventional laser therapy.

The drug therapy resulted in mild anatomical retinal abnormality in just one eye of 31 infants, whereas conventional laser treatment resulted in a mild structural abnormality in 16 eyes and severe abnormality in two eyes of 33 infants.

“We’ve gone from nothing to a real solution. It you are careful and administer this therapy appropriately in stage 3+, you can get wonderful outcomes,” said Mintz-Hittner, the study’s principal investigator.

Mintz-Hittner, an attending physician at Children’s Memorial Hermann Hospital and the Robert Cizik Eye Clinic, stressed that timing is critical with this drug therapy. If administered too early, in stages 1 and 2 of the disease, it can cause retinal dystrophy. Given too late, in stages 4-5 of the disease, the drug can accelerate retina detachment.

“With this drug therapy, we use a few drops of anesthetic to numb the eye. We take a syringe with a tiny needle and administer a small amount of the drug directly into the eye. The whole process takes two to three minutes, and you begin to see results within 24 hours,” Mintz-Hittner said.

“The abnormal vessels virtually disappear and then normal vessels begin to grow out again. The field of vision is preserved and myopia is less.”

The study has been published in the New England Journal of Medicine. (ANI)