Clinical Trials for Marfan Syndrome to begin at Brenner Children's Hospital
WINSTON-SALEM, N.C. – Pediatric researchers at Brenner Children’s Hospital will be testing a new medication for Marfan syndrome. Wesley Covitz, M.D., head of pediatric cardiology, and his team will test whether a drug commonly used to treat high blood pressure will be effective in children and young adults with Marfan Syndrome and will compare their findings with results of the currently used beta-blocker medication.
Patients will be given the original beta blocker or an angiotensin receptor blocker (the new medication) to slow the enlargement of the aorta (a large artery which carries blood away from the heart) which can result in sudden tearing of the aorta if left untreated. Both drugs are commonly used to treat high blood pressure in adults and the new drug has been found effective in laboratory studies, Covitz said.
Marfan syndrome is a relatively rare genetic condition in which patients lack a gene that allows them to make elastic tissue. Patients with the disease tend to be extremely tall, often 6 feet 5 inches or taller. They have vision problems, long arms and fingers and often have scoliosis, Covitz said.
For more than 20 years, Brenner Children’s Hospital has had the only regional referral multi-disciplinary clinic in the Southeast dedicated to patients with this syndrome.
“The children’s hospital houses the only multi-disciplinary program in the state providing expertise in the treatment of Marfan syndrome,” Covitz said. “We’ve had several patients who were unaware they had the disease until they were diagnosed at age 40. The disease can be fatal if it’s left untreated, however with the proper treatment, many patients can live a normal life span.”
If a parent has the disease, there is a 50 percent chance that their children will inherit the disease, Covitz said.
Brenner Children’s Hospital will be participating in this National Institutes of Health clinical trial with nine other children’s hospitals around the country, enrolling over 400 children and young adults. The children will be followed closely for a year to 18 months.
Brenner Children’s Hospital is one of eight centers in the United States participating in the National Institutes of Health (NIH) Pediatric Heart Network, which conducts clinical trials on patients with heart defects.
“This is a real honor for Brenner Children’s Hospital,” said Covitz. “This allows us to merge resources with other prestigious academic medical centers and find ways to improve care for children with heart problems. By conducting clinical trials, we are able to offer our patients the very latest treatment advances, placing them on the road to recovery with better outcomes.”
This clinical trial is sponsored by the Pediatric Heart Network and the National Marfan Association and will begin in October. For more information about the children’s hospital, visit www.brennerchildrens.org.
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Wake Forest University Baptist Medical Center is an academic health system comprised of North Carolina Baptist Hospital, Wake Forest University Health Sciences and Brenner Children’s Hospital. The system comprises 1,187 acute care, rehabilitation, psychiatry and long-term care beds and is consistently ranked as one of “America’s Best Hospitals” by U.S. News & World Report. Brenner Children’s was named one of the top children’s hospitals in the nation by Child magazine.
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