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AN AMBITIOUS STUDY AIMS TO UNDERSTAND HOW CROHN'S DISEASE STUNTS KIDS' GROWTH

Eventually, Alex's parents brought him to NewYork-Presbyterian/Weill Cornell Medical Center, where he saw Dr. Thomas Ciecierega, an assistant professor of pediatrics. Dr. Ciecierega made the correct diagnosis — Crohn's disease, a type of inflammatory bowel disease (IBD) that is characterized by intestinal inflammation — and put the boy on the proper course of medication. "He started gaining weight and was more active," Caroline says. "You could see the life coming back." Today, Alex is still a little short for his age, but the Long Islander is an active 9-year- old and avid lacrosse player. He'll have to stay on medication indefinitely, and the disease has other potential complications, like eye problems and arthritis. But for now, he's doing well. Says his mom: "This is the healthiest I've ever seen him in his life."When Alex Bancroft was 5, it was clear that something was awry with his digestive system. During kindergarten, his mom says, he was spending a lot of time in the bathroom. Doctors first thought he had a virus, but the symptoms persisted. Then a pediatric gastroenterologist diagnosed Alex with irritable bowel syndrome and put him on medication for it. But he didn't get any better — and on top of enduring diarrhea and fatigue, he wasn't growing. "He wouldn't eat, because he was afraid that he wouldn't make it to the bathroom in school," says his mother, Caroline Bancroft. "At age 7, he dropped down to about 40 pounds."

Someday, kids like Alex could benefit from research now under way at Weill Cornell Medicine. Dr. Neera Gupta, director of research and a specialist at the Weill Cornell Medicine Pediatric Inflammatory Bowel Disease Center and an associate professor of pediatrics, recently launched a study to uncover why kids with Crohn's — especially boys — are often shorter than their peers. Growth is a dynamic marker of a child's health status, she notes, and it's an issue of central importance to patients, families, and providers.

Dr. Gupta, who is also a physician-scientist at the Jill Roberts Institute for Research in Inflammatory Bowel Disease at Weill Cornell Medicine, notes that roughly 25 percent of patients with IBD are diagnosed during childhood and adolescence. In the United States, there are about 5,000 new pediatric IBD cases each year — and they present unique challenges. Not only is growth impairment a major complication of pediatric-onset Crohn's disease that distinguishes it from the adult-onset version, she explains, but diffuse disease is more common in children at the time of diagnosis, and progression occurs more frequently in kids than in adults.

There are also sex differences in the presentation and course of Crohn's disease. In Dr. Gupta's previous studies, she found that girls tend to have a more severe course of disease and are more likely to need surgery, but boys are more likely to have growth problems. Her current work, funded by an NIH award of more than $3 million, aims to figure out why.

The study will follow children and adolescent boys and girls for two years. After an initial screening to make sure they qualify, participants will come in every six months for a total of five visits, either at Weill Cornell Medicine or one of the other collaborating institutions. Procedures will include height and weight measurements, assessment of pubertal status, medical history, blood draw, hand x-rays (for bone age) and nutrient intake assessments.

Dr. Gupta's team will utilize the data to develop predictive models to identify which children are most likely to have persistent growth problems caused by Crohn's disease, ultimately helping clinicians to better determine a course of treatment. "One of the debates in our field is when to introduce the most aggressive therapies for Crohn's disease," Dr. Gupta says. "Some believe we should do it at the time of diagnosis, and some believe we should start with other therapies first. Most agree there are high-risk children who would benefit from early intervention with aggressive therapy — but we need to figure out who these high-risk children are. Because there is a very narrow therapeutic window in which we can intervene to improve growth, children who are at highest risk for persistent growth impairment may be candidates for early intervention with aggressive therapy."

After data collection is completed, doctors will be able to look at a combination of variables — including sex, race/ethnicity, disease location, bone age, inflammatory markers, hormone levels and genetic markers — to determine whether a child is at high risk for growth impairment. The next step will be an interventional clinical trial assessing aggressive therapies in children who are at highest risk for remaining growth-impaired. Importantly, Dr. Gupta says, the study will improve understanding of the underlying mechanisms of growth impairment in Crohn's disease. It's known that impairment may result from inflammation, malnutrition, or the side effects of medications; despite this knowledge, it continues to occur frequently in children and adolescents. "We need to advance our understanding of the underlying mechanisms of growth impairment in Crohn's disease," Dr. Gupta says. "We hope that findings from our research will lead to improved treatments not only for the children identified as high-risk for persistent growth impairment, but for all children with IBD."

Dr. Gupta aims to complete data collection by 2019, but for now her team is still in the process of recruiting participants. The goal is 125 — but that's challenging, in part because not every Crohn's patient in the specified age range will qualify for the study. Alex, for example, hasn't qualified so far, because his bone age hasn't met the eligibility criteria. But his parents hope that work like Dr. Gupta's will someday ensure that other kids don't have to go through the ordeal that he has. "Ultimately," says his dad, Bob Bancroft, "what we're hoping for is a cure."

— Keri Blakinger

This story first appeared in Weill Cornell Medicine, Vol. 15, No.2.

Posted September 9, 2016 11:00 AM | Permalink to this post

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