Amy Ray loves toys, gymnastics, reading and coloring. The 6-year-old dreams big, takes care of her pets and enjoys adventures on her family's boat.
"I like to go really fast," this active first grader says.
With every new passion Amy Ray discovers, her parents encourage her.
Her mom and dad, Kyle and Brooke, fell in love with Amy Ray when she was just 33 days old. Before they adopted her at 18 months, they received some unexpected news.
"When she was close to 1, we found that there were issues with her liver enzymes," Brooke says.
Their local pediatrician ordered a blood test and discovered that Amy Ray had hepatitis C.
What is hepatitis C?
Hepatitis C is a virus that causes the liver tissue to swell and become inflamed. It spreads through contaminated blood and can be passed from an infected mother to her baby.
"We were worried," Brooke says. "We knew it wasn't something that would affect her immediately, but we also knew that later on, she was at risk for complications."
In contrast to hepatitis A and B, there is no vaccine for hepatitis C. The approved drug therapy for younger children, with pegylated interferon and ribavirin, has many serious side effects and it is not effective in many of the patients. A new group of direct-acting antiviral drugs against hepatitis C has shown great success in adults and minimal side effects.
From the day Amy Ray became a part of their family, Kyle and Brooke searched for a treatment for their daughter. Now, thanks to a recent clinical trial at UT Southwestern Medical Center in conjunction with Children's Health℠ Gastroenterology, that search is over.
"Whatever challenges Amy Ray faced, we knew we wouldn't outgrow the capabilities of Children’s Health," Kyle says.
Norberto Rodriguez-Baez, M.D., Gastroenterologist, and Associate Professor, UT Southwestern, is internationally recognized for his hepatitis research. He's made it his mission to find a cure for children like Amy Ray. In search of a cure, Amy Ray's family came to Children's Health shortly after she was diagnosed to work with Dr. Rodriguez-Baez and his team.
"The hepatitis C virus has been very difficult to treat," Dr. Rodriguez-Baez says. "In pediatrics, the disease typically progresses very slowly, however, it can lead to serious complications such as scarring of the liver and cancer."
Some of side effects for the current hepatitis C treatment in children include nausea, fatigue, decrease in platelets and white blood cell counts and delayed growth patterns. In addition to that, in Amy Ray's case, Dr. Rodriguez-Baez said the chance for effectiveness was slim because of her genotype. The Roberts decided to wait and hope for a better option.
Groundbreaking study brings new hope
In 2014, Dr. Rodriguez-Baez was chosen to participate in a national, multi-center study at Children's Health, testing the safety and effectiveness of the combination of two direct-acting antiviral medications among three different age groups: 0-2, 3-11 and 12-18.
Once UT Southwestern and Children's Health started looking for clinical trial patients, Amy Ray came right to the physicians’ minds. Allison Johnson, Clinical Research Coordinator at Children's Health, says Amy Ray’s energy inspired them.
"Amy Ray is always very excited," Allison says. "She keeps you on your toes and she's brave."
After four years of waiting and a series of tests, Amy Ray's family received the news they had been holding out for: She was selected as one of the five patients to participate in the clinical trial and started the 12-week treatment trial in July 2017.
Kyle was thankful for the team's guidance through the process. "Allison always broke it down to help us understand what we were doing, where we were going and what was going on, which was really comforting," Kyle says.
Filled with hope, Amy Ray's family drove in frequently from Melissa, Texas, for checkups, meetings and even a 14-hour study day. They say the Children's Health team worked tirelessly to care for their every need.
"Allison would take calls at all times, meet us early so we could get there before traffic and make it all work," Kyle says. "Andy was always there to keep Amy Ray entertained, bringing her coloring books, games, anything to make her smile."
"The best news ever"
Dr. Rodriguez-Baez says the trial used a combination of two antiviral drugs, testing to see how they worked together to eliminate the virus. After three months of post-trial checkups, Amy Ray's family, as well as all the other trial patients, got the news they dreamed of hearing.
"Hearing that she was cured, it was amazing," Brooke says. "It was the best news ever."
"This is a big deal," Dr. Rodriguez-Baez says. "In the past, this virus was very difficult to treat and cure, but now, we are able to treat and cure these children."
Due to this study, in 2017, the Food and Drug Administration approved this regimen for adolescents 12 and older and is proving effective. The data from Amy Ray's age group was submitted and approvals are pending.
"Being able to engage in clinical trials to provide hope for families and see their happy faces, that's one of the most fulfilling things a physician can experience," Dr. Rodriguez-Baez says.
Ranked among the top pediatric gastroenterology programs, our pediatric GI team offers minimally invasive diagnostic techniques and the latest innovative treatments delivered with a personalized approach that prioritizes peace of mind. Learn more about our pediatric liver disease program.
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