Monday, November 23, 2015
Frustrated by the lack of answers, a Bahamas family traveled to Cincinnati Children’s Hospital Medical Center to find out why 3-year-old Brayden Newbold’s growth stalled shortly after he was born.
"When he was 6 months old, our pediatrician told us his growth was minimal and falling off the growth chart. After a series of tests, we spent the next year and a half not knowing what was wrong,” said Brittany Newbold, Brayden’s mom.
A family friend in Spanish Wells, Bahamas, suggested the Newbolds contact Philippe Backeljauw, MD, clinical director of the Cincinnati Children’s Growth Center in the Division of Endocrinology. He diagnosed Brayden with Laron Syndrome, a rare genetic growth disorder that causes short stature and low blood sugar.
"His disorder is better called 'growth hormone resistance’ which means that he makes plenty of growth hormone but it doesn't have a good biological effect,” said Dr. Backeljauw. “This disorder of growth hormone resistance leads to severe deficiency of a growth factor called IGF-1 or insulin growth factor 1.”
In the 1990’s, researchers at Cincinnati Children’s conducted a study for a drug called “Increlex” for treatment of patients with Laron Syndrome. Patients from all over the world took part in the study. The Food and Drug Administration approved the drug for patients with the IGF-1 deficiency in 2005.
“The hope is that the medication will remain available and we can continue to treat these patients until adult height which will make a tremendous difference for them,” said Dr. Backeljauw.
When Brayden arrived at Cincinnati Children’s, his height was closer to a 1 ½ year old. For the last nine months, his parents have given him Increlex injections, twice a day, every day. Brayden has grown 3 ½ inches during that time. Since the drug manufacturer won’t ship the medication to the Bahamas, the Newbolds travel to Cincinnati Children’s every three months.
“It’s worth it to see how remarkable he is doing,” said Newbold. “He’s running and climbing, which he wasn’t before. He’s keeping up with the other kids since he started treatment. It’s given him that confidence he needs.”
Cincinnati Children’s Hospital Medical Center ranks third in the nation among all Honor Roll hospitals in U.S. News & World Report’s 2015 Best Children’s Hospitals. It is also ranked in the top 10 for all 10 pediatric specialties, including a #1 ranking in pulmonology and #2 in cancer and in nephrology. Cincinnati Children’s, a non-profit organization, is one of the top three recipients of pediatric research grants from the National Institutes of Health, and a research and teaching affiliate of the University of Cincinnati College of Medicine. The medical center is internationally recognized for improving child health and transforming delivery of care through fully integrated, globally recognized research, education and innovation. Additional information can be found at www.cincinnatichildrens.org. Connect on the Cincinnati Children’s blog, via Facebook and on Twitter.