Retinopathy of Prematurity (ROP) Pediatric Research and Clinical Trials

As advances in neonatal care make it possible to save more premature infants than ever before, doctors and researchers face a growing challenge in diagnosing and treating preemies’ unique health problems. Retinopathy of prematurity is one of the most common of these problems and, while often mild, does carry the risk of blindness in some children. Children’s Hospital Boston is working to preserve the gift of sight in all babies by exploring new ways to diagnose, treat and possibly prevent ROP and its consequences.

Weight gain and ROP risk

The ability to predict which premature babies will develop serious ROP would not only give doctors a vital head start on treatment, but also prevent costly and unnecessary testing on infants who aren’t at risk. Along with gestational age and weight, one of the strongest predictors of ROP is poor weight gain during the first weeks of life. Working with colleagues overseas, Children’s ophthalmologists have found that by tracking the weight gain of preemies, they can accurately predict which ones will develop ROP serious enough to require treatment.

Moreover, their tracking system—a computer alogrithm called WINROP—can identify these babies several weeks sooner than current screening methods allow, offering new opportunities to intervene early and possibly even prevent serious disease from developing.

New treatment targets

Current treatments for ROP, like photocoagulation and cryopexy, aim to halt the spread of abnormal blood vessels by burning or freezing small portions of the eye. In search of less invasive therapies, Children’s doctors and researchers are exploring how medications or even dietary supplements might play a role in combating ROP. Among their areas of interest:

  • Insulin-like growth factor 1 (IGF-1):Babies first receive this substance, which is critical to blood vessel growth in the eye, from the amniotic fluid. But because they are born early, premature infants tend to have lower levels of IGF-1, which is associated with higher ROP risk. Medications that can boost IGF-1 levels into the normal range are now in development.
  • Vascular endothelial growth factor (VEGF): VEGF is another important substance that’s first received in utero, but its role in ROP is more complex: Too little VEGF inhibits normal vessel growth; too much can fuel the growth of abnormal vessels. Medications that can regulate VEGF (like the new drug Avastin, which blocks its actions) may help doctors correct these harmful imbalances.
  • Omega-3 polyunsaturated fatty acids:Mice studies have shown that these fatty acids, found in popular fish oil supplements, help protect against blindness caused by abnormal blood vessel growth in the eye. Children’s researchers are now investigating whether these omega-3 fatty acids are equally effective in infants.
    Omega 3’s and retinopathy

    Lois Smith, MD, PhD of Ophthalmology at Children’s led research investigating the role of omega-3 fatty acids, which is commonly found in fish, in helping to prevent retinopathy. Smith found that omega-3 promote the growth of healthy blood vessels in the eye. Learn more about this study in the Children’s newsroom.

Clinical trials

There are many ways in which your child might benefit from Children’s Hospital Boston’s medical research program. Children’s doctors and scientists have made many breakthrough discoveries about diseases like polio and leukemia; our ongoing innovative research continues to push the boundaries of the way pediatric medicine is practiced.

It’s possible that your child will be eligible to participate in one of Children’s current clinical trials. These studies are useful for a multitude of reasons: Some trials are designed to evaluate the effectiveness of a particular drug, treatment or therapy on a specific disease; others help doctors to better understand how and why certain conditions occur. At any given time, Children’s has hundreds of clinical trials under way.

And participation in any clinical trial is completely voluntary: We will take care to fully explain all elements of the treatment plan prior to the start of the trial, and you may remove your child from the medical study at any time.

To search current and upcoming clinical trials at Children’s, go to:

To search the NIH’s list of clinical trials taking place around the world, go to: