Premature babies born with severe forms of the potentially blinding eye condition retinopathy of prematurity (ROP) should be treated promptly after diagnosis because they continue to benefit from early therapy well into their preschool years, according to a nationwide study conducted at Johns Hopkins Children’s Center and 25 other pediatric hospitals.  

ROP, a disorder affecting about 15,000 U.S. babies each year, is marked by abnormal growth of blood vessels in the eyes that causes scarring and detachment of the light-collecting retina.  

The new study, to appear online on April 12 in the Archives of Ophthalmology, builds on research published by the same group in 2003, which found that earlier surgery in infants to remove abnormal blood vessels improved outcomes to age 2 in the youngest children with severe forms of the disease, defined by dilated blood vessels in the retina growing in a twisted pattern. Such children are treated with laser surgery or cryotherapy (freezing), both of which involve destroying the chaotically growing blood vessels to prevent further retinal damage and vision loss. Until the first study, however, the standard treatment for such infants was to hold surgery until the risk of retinal detachment reached 50 percent, but with early treatment, children with severe forms of the disease undergo surgery before they reach the 50 percent threshold. 

In the second phase just reported, the researchers followed 370 of the original 401 patients through age 6 and not only confirmed the early findings, but also showed that high-risk children continue to do well at age 6 if they received the early treatment.  

The team also confirmed results from the first study that children with milder forms of ROP do not benefit from early treatment and more than half improve without it. These findings, the researchers say, underscore the importance of careful monitoring rather than aggressive treatment of those low-risk children. 

“Our new findings have answered the most vexing question — do the benefits persist — after the original study and in doing so further highlight how critical it is to tailor treatment decisions to each individual patient’s risk and overall medical condition,” said study investigator Michael X. Repka, M.D., of the Johns Hopkins Wilmer Eye Institute  and deputy director of ophthalmology at Johns Hopkins Children’s.  

In the follow-up study, nearly 75 percent in the high-risk group had good visual acuity after early treatment, compared to 67 percent among those who received standard treatment. Also, 15 percent of high-risk eyes treated under the standard protocol had substantial retinal damage, compared with only 9 percent of high-risk eyes treated early. 

These findings should help pediatric ophthalmologist move away from one-size-fits-all medicine toward more individualized therapy, the investigators say. 

“The long-term study has given clinicians evidence that infants with ROP should be treated with different strategies based on an infant’s risk for a severe form of the disease, which can be determined through an exam at the bedside,” said lead investigator William V. Good, M.D., of Smith-Kettlewell Eye Research Institute in San Francisco.  

Investigators caution that not all children who qualified as high risk benefited from early therapy, and some of them developed vision loss despite their treatment. The finding emphasizes the critical importance of ROP prevention, the researchers say, because even with proper and timely treatment, some children do not do well.  

The majority of babies born with ROP have a mild form of the disease that does not require treatment, but those with more severe forms tend to develop visual impairment, even blindness. Up to 600 infants become legally blind each year in the United States because of ROP, the investigators estimate.  

The research was funded by the National Eye Institute.