What's new in the treatment of retinopathy of prematurity?

Answers from Robert V. Johnson, M.D.

An anti-cancer drug, bevacizumab (Avastin), is shaping up to be an effective alternative to laser surgery in retinopathy of prematurity (ROP), an eye disease that affects many premature babies. However, more research is needed before doctors understand the full effects of bevacizumab on preterm infants.

Retinopathy of prematurity (ROP) generally affects preterm infants born before week 31 of pregnancy and weighing 2.75 pounds (1,250 grams) or less at birth. In most cases, ROP resolves without treatment, causing no damage. Advanced ROP, however, can cause permanent vision problems or blindness.

In ROP, blood vessels swell and overgrow in the light-sensitive layer of nerves at the back of the eye (retina). When the condition is advanced, the abnormal retinal vessels extend into the jelly-like substance (vitreous) that fills the center of the eye. Bleeding from these vessels may scar the retina and stress its attachment to the back of the eye, causing partial or complete retinal detachment. Laser surgery, the standard treatment for advanced ROP, saves sight in the main part of the visual field, but at the cost of side (peripheral) vision. Laser surgery also requires general anesthesia, which may be risky for preterm infants.

Bevacizumab has Food and Drug Administration approval for treating colon cancer. The drug works by blocking the growth of blood vessels that tumors generate to sustain themselves. Bevacizumab is also widely used to curb the overgrowth of retinal blood vessels in two serious adult eye diseases, wet macular degeneration and advanced diabetic retinopathy. The drug has shown some promise in treating ROP in initial research and may be an option for preterm infants at highest risk of vision loss.

More research is needed into the timing of the medication for a preterm infant, the optimal dose of the medication and how long its effects last. Doctors don't yet know the long-term impact of using this drug in preterm infants. Although bevacizumab doesn't seem to leak out of the retina, some concern exists that the drug might slow down the formation of normal blood vessels in other parts of a baby's body.

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Robert V. Johnson, M.D.

Oct. 10, 2014