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One big question:  ten diligent years

Emory Eye magazine, Summer 2010, pp. 2-7.

by Ginger Pyron

 

And hundreds of people—doctors, clinical coordinators, biostatisticians, parents—who, led by the Emory Eye Center, have been daily collecting evidence that will answer: For an infant born with a cataract in one eye, which treatment works best?  This question, raised by Scott R. Lambert, pediatric ophthalmologist at Emory and both the principal investigator and study chairman of the 2004-2014 nationwide Infant Aphakia Treatment Study (IATS), sounds simple.

Arriving at a conclusive answer, however, requires years of meticulous research. Twelve eye institutes throughout the United States have risen spectacularly to the challenge.

A vision-threatening condition

If the word “cataract” makes you think of Niagara Falls, you’re on the right track. Originally meaning a cascade of water, it also describes the milky white area that doctors notice in the eyes of some 300 infants in the United States each year who are born with a unilateral congenital cataract.

Instead of the eye’s usual transparent lens, which focuses objects onto the retina so they become visible, these babies have a cloudy, opaque lens that doesn’t allow light to pass through to the retina. Babies with this condition cannot see out of the affected eye.

Kimberly Burkett and Lacey Weeks—the mothers of Emory IATS patients five-year-old Marvin, Jr. (M.J.) and three-year-old James, respectively—recall the day, years back, when they first learned about their sons’ vision problem:

Kim: M.J. wasn’t even two months old. In a routine follow-up, our pediatrician noticed something unusual in M.J.’s eye and immediately referred us to a children’s hospital. We learned that M.J. had a cataract and needed surgery—soon. The next day, we were in the car driving to Emory.

Lacey: My husband and I didn’t even know that children could be born with cataracts. At James’ four-month checkup, our doctor said, “I’m going to get you an appointment with the ophthalmologist right now.”

There’s good reason for the urgency. If the cataract isn’t surgically removed within a few months, the young eye will not develop properly and the child’s vision can become permanently damaged.

Removing the cataract creates a new condition, called aphakia (from Greek a + phakos, “no lens”). So after surgery, that eye will still need help. For infants, the standard treatment has been a contact lens; increasingly, ophthalmologists have treated older children by implanting an intraocular lens (IOL) during the cataract surgery, then prescribing glasses for residual correction. In either case, to ensure that the treated eye keeps learning how to focus, doctors prevent the child’s other eye from helping out, by covering it with an eye patch.

Both treatments are safe and work well. But over the long term, which one best supports optimal visual acuity?

A model study

M.J. Burkett and James Weeks became patients in the IATS trial, which has treated 114 babies across the United States. Funded by the National Eye Institute (a branch of the National Institutes of Health) and planned to span a decade, the study is now in its seventh year.

Dr. Lambert and Emory’s Lindreth (Lindy) DuBois, senior associate in ophthalmology, who serves as the national coordinator, oversee the entire project, making sure that at each of the 12 IATS centers, every step is conducted in exactly the same way. The rigorously standardized process sets this project apart and gives the study its exemplary reliability. Each center also has its own site coordinator. Rachel Robb, who fills that role at Emory, says, “No one has ever done a study like this before, on this scale. In the future, when a baby is born with a cataract, we’ll have a better way, a safer way, to treat that child.”

Another strong asset for Emory is the university’s own Rollins School of Public Health, specifically the Department of Epidemiology and the Data Coordinating Center in the Department of Biostatistics and Bioinformatics. “These academic groups have helped with study design and data analysis,” DuBois explains. “Right here on campus, we have the experts who are actually collecting and crunching the data.”

Starting in 2004, from Miami to Portland, Ore., from Dallas to Boston, and at other sites in between, PIs and coordinators carefully recruited patients who matched the study’s criteria. Because IATS is randomized, parents had to be willing not to know which of the two treatments their tiny baby—between four weeks and seven months old—would receive. Even the surgeons did not know, until the day of each surgery, whether they would be simply removing the cataract and prescribing a contact lens, or removing the cataract and implanting an IOL.

Throughout, the study is being monitored by numerous regulatory groups: NEI’s Data Safety Monitoring Committee, the Food and Drug Administration, and each site’s own Institutional Review Board. These groups interact with each other, scrutinizing the process in terms of patient safety and ethics. “A lot of people are observing this study,” says Dr. Lambert, “making sure that everything is in perfect order.”

Working together

For the parents of M.J. and James, the study has progressed smoothly. Their sons’ surgeries, here at Emory, went well, with both boys receiving an IOL, glasses, and a supply of eye patches. The families enjoy close relationships with Rachel Robb, whose ongoing roles include consultant, coach, and cheerleader. Four times a year (twice a year after age five), the families return to Emory for follow-up visits. For the Burketts, who live in Jacksonville, Fla., that means a 700-mile round trip, contained in a single day.

Kim: M.J. and I have the trips down to a perfect routine; he’s a veteran now. And I don’t mind driving a long way for my son. Emory’s the best, so that’s where I want him to be. Everything has been a plus: I love getting the newsletters. I love Rachel.

Lacey: We’re glad that James could go to a top-notch hospital and participate in a study with one of the top doctors. Dr. Lambert is wonderful. And Rachel is so understanding. She makes sure we’re top priority.

The appreciation goes both ways. “The families are the heroes of this study,” Robb says. “We ask a lot from them: all the appointments, the questionnaires, the documentation. And the constant patching. It’s hard work.”

Kim: The patch is a part of our life. For years, as soon as M.J. woke up, I’d say, “Good morning, brush your teeth, put your patch on.” His sister Kai wore a patch, to support him. We put a patch on his teddy bear, on his dad. Now that M.J. is five, he only has to patch two hours a day. We use a little timer, and he’s so excited when it finally beeps.

Lacey: Just before age one, James figured out, “Hey, I can take this off!” Year two was the biggest struggle. The IATS newsletter has given us good ideas: patch while eating, set up a chart with stickers. When we’re reading books, we always patch. The goal is to patch half of his waking hours, which is hard at this age. But it’s become second nature. We patch; that’s what we do.

Robb emphasizes that all these families would have received treatment for the babies’ cataracts even had they chosen not to enroll in the study. Participating in IATs requires a huge commitment of time and effort; in return, the children receive the highest level of individual care and attention by multiple specialists year after year.

Noteworthy outcomes

In December 2009, each site completed visual acuity testing on its patients at age one. During its five-year extension, the study will test all the participants until they reach age five, when standard visual acuity testing can be performed.

Key players in IATS deem the project a remarkable success. Dr. Lambert cites particular triumphs: “First, 80 percent of the families who were eligible to be in the study agreed to participate, which is a very high rate. Furthermore, we were able to test every child in the study at one year of age. That’s extremely rare, and it reflects the relationships our coordinators have established with these families.”

DuBois attributes the study’s achievements to “an incredible network of efficient and caring eye practitioners. With this study, Emory has set an enviable standard of documentation and teamwork. Dr. Lambert now has a machine in place that he can use for other studies.”

According to Dr. Lambert, “The whole world is watching this study. Treatment for infant aphakia is an issue that people are asking about in every country we visit, because most countries don’t have the resources to do this kind of trial. So our work will affect children throughout the world.”

Kim: Overall, M.J. has been so good through the whole process. I give him a lot of credit. Now we’re eager to hear about the results. As far as our part goes with M.J., they’re going to be accurate. We’ve dotted every i and crossed every t.

Lacey: James just turned three, and he loves putting his glasses on and looking at himself in the mirror. Watching him, I’m very grateful for this study. I feel proud, knowing that our family is benefiting others who’ll have to deal with this same condition.

Here at the Emory Eye Center, we’re feeling proud, too.

Sharing the knowledge

The carefully gathered, many-layered data from IATS is already shaping further research. A preliminary paper on the study was published in January; the main report—containing the much-anticipated results from the testing of one-year-olds—will appear online in May, in print during July. Numerous ancillary papers will follow, covering particular facets of the study.

“In a workshop this past April, at the annual meeting of the American Association of Pediatric Ophthalmology and Strabismus,” says Dr. Lambert, “we shared videos from some of the surgeries, which are helping us make this knowledge broadly transferable.”

Emory Eye Summer 2010Emory Eye magazine, Summer 2010 [download a pdf copy].

BREAKING NEWS | Findings for the IATS study were released prior to printing of the magazine.  Results show that there is not a significant difference in visual acuity in children, whether the child is fitted with a contact lends (CL) or given an intraocular lens (IOL) immediately following cataract removal.  Testing these children at 1 year of age resulted in similar visual acuity outcomes for both groups.

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