Name: Zachary McCarthy and his parents
Home State: California

Zachary has been participating in clinical research since 2001

Why? Zach's parents wanted to give Zach the chance for a less severe outcome from retinopathy of prematurity (ROP) — a blinding disease that affects premature, very low birthweight infants — and to help other premature infants in the future

The McCarthys' story: "Mommy, what's an extinguisher?" asked 5-year-old Zach, after passing a fire extinguisher in the local shopping mall. It was a strange word for so young a child to be reading, thought his mother, Michele. What made it all the more amazing is that Zach spent the first 4½ months of his life in a neonatal intensive care unit, in a fight for life so unlikely to be won that the nurses later called him their "miracle baby." His eyesight had been at particular risk.

Born 15 weeks early, Zach weighed only 2 pounds 1 ounce. He was the size of a Cornish game hen, as his uncle put it. "His lungs weren't developed," says Michele. "His eyes were still fused shut. He was so delicate his skin appeared translucent. We named him Zachary, which means 'God remembers.' "

Zach's prematurity caused problems with his eyes, lungs, and kidneys. By the time he was 4 months old, he'd undergone five surgeries. One complication that needed treatment was retinopathy of prematurity, or ROP, a potentially blinding eye disorder that affects infants born very prematurely.

ROP is unpredictable. Blood vessels in the eye stop growing or take a wrong turn. Abnormal vessels leak fluid and blood and can cause scarring, increasing the risk of retinal detachment, severe vision loss, or blindness. Dr. William Good of the Smith-Kettlewell Eye Research Institute in San Francisco talked with Zachary's parents about participating in a study sponsored by the National Eye Institute at NIH.

The NIH study was designed to provide important information about treatment options and timing. "Somewhere in the disease process a point is reached beyond which there is no turning back," says Good. The hypothesis in the ETROP study was that performing surgery before that threshold was reached could prevent a more severe form of ROP.

The McCarthys faced a scary decision: Should they let Dr. Good stop abnormal blood vessel growth by using a laser to burn the edge of the retina - before Zach's eyes had reached that threshold? The elective surgery (which might not be necessary if Zach never reached the threshold) would destroy good tissue, resulting in some loss of Zach's peripheral vision. But the alternative - possible blindness - was worse. The probability of the need for surgery was high, but was not confirmed. The surgery would help confirm if early elective surgery was the right path. Calmly and patiently, Dr. Good answered the McCarthys' questions, addressing their need to understand both clinical and emotional risks.

"We decided we had to go for treatment," recalls Michele. "We also decided to be part of this national study because it may help other children." The research will give doctors better information about when and how to treat ROP.

"It is crucial that infants with high-risk ROP be identified early and be given timely treatment," says Dr. Good, the study's chair. " We learned from this study that earlier treatment could save infants from a lifetime of visual impairment. The results also clearly indicated that for certain subgroups of eyes, watchful waiting and not immediate treatment is the best approach." The study will follow patients like Zach until age 6 to ensure that the benefits of early treatment persist into childhood. "Because visual acuity continues to develop during infancy and early childhood, the long-term effect of early treatment on visual development is not yet fully known, says Dr. Good. "We expect that the significant benefits to vision found in this study will persist into childhood, but we have to be sure."

In Zach's case, Dr. Good did surgery first on one eye (the test eye) and then a week later, when the second eye reached the threshold, did surgery on the other eye (the control). Zach is still part of the research.

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