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A new trial at St. Jude helps children with chronic ITP, a rare bleeding disorder marked by low platelet counts.
Like most teenage boys in his rural community, Wesley Tice-Gray loves to hunt, fish and roar through the woods on an all-terrain vehicle. Three years ago, he ended up in the hospital after one such jaunt. Ironically, Wesley’s injury occurred while the vehicle was stationary.
“I was just getting off the four-wheeler when my foot slipped and the handlebar caught me in the stomach,” Wesley recalls. “A big bruise came up.”
By late afternoon, the bruise was larger than a softball and as ominous as a thundercloud. Concerned, his family took Wesley to the doctor and then to the hospital. Blood tests indicated that the teen’s platelet count was dangerously abnormal. Produced by the bone marrow, platelets are small cells that help the blood clot. Instead of having 150,000 to 450,000 platelets per microliter of blood, Wesley had only 2,000.
Doctors discovered that Wesley had immune thrombocytopenia, or ITP, a bleeding disorder that affects about 3,000 children each year in the United States. Wesley’s immune system was waging war on his platelets—coating them with antibodies and marking them for destruction. Without an adequate number of platelets, Wesley was at risk for bleeding, bruising and other more serious complications.
In retrospect, Wesley’s family realized that the symptoms of ITP had been present for years.
“I had started noticing bruises when he was in about the third grade,” says his grandmother, Becky Tice. “But I thought he was just being a typical kid.”
“He has always been active,” adds Wesley’s aunt, Susan Bing. “Most kids are clumsy. We thought, ‘He’s just being a boy, out playing and running into things.’”
Children with ITP usually have an acute form of the disorder, which resolves after a few weeks or months. But Wesley had chronic ITP, a form that rarely affects children or adolescents and can persist for years. The clinicians immediately referred him to St. Jude Children’s Research Hospital, where hematologists could provide the specialized treatment he required.
At St. Jude, Wesley met hematologist Jenny McDade, DO. McDade explained that antibodies in Wesley’s body were adhering to newly formed platelets, incorrectly flagging them as foreign invaders. As part of its quest to eliminate disease-causing cells, the spleen recognized the antibodies as signs of enemy infiltration and eradicated the platelets.
In addition to large bruises, many children with ITP have nosebleeds, blood in the urine and small pink dots called petechiae on their skin. Their gums may bleed when they brush their teeth, and a head injury that would be minor for most children can be life threatening.
Most traditional ITP treatments typically improve platelet counts for several weeks, but do not cure the underlying disease process. The side effects can be significant, commonly including headache, fever, chills, nausea and vomiting. Rarely, more serious side effects can occur. Children undergoing treatment often require hospital admission.
Patients like Wesley with chronic ITP are forced to live with the limitations and lifestyle changes that accompany extremely low platelet counts.
“Unless children develop bleeding symptoms, we typically don’t expose them to the potential side effects of the available treatments, because we know the effect will wear off within a few weeks,” McDade explains. “Basically, traditional treatments don’t stop the development of antibodies or the coating of platelets with those antibodies. Rather, the medications just temporarily prevent the antibody-coated platelets from being destroyed.”
Researchers have been seeking a way to encourage the bone marrow to increase platelet production so that more platelets would circulate through the blood. One such drug recently received FDA approval for adults with chronic ITP. McDade was excited. Would this medication also help children with the disorder?
About a year ago, St. Jude obtained access to the new medication, called eltrombopag, through participation in an international clinical trial. Through the study, eltrombopag would be offered to 70 children worldwide. This study would be the first pediatric trial for the agent. The protocol would be open to children for whom existing ITP treatments had not worked and who still had dangerously low platelet counts.
Wesley leapt at the opportunity to take eltrombopag for his chronic ITP.
“I was the first kid in the U.S. and the second in the world to get the new drug,” Wesley brags. “Some kid in Europe beat me to it, or I’d have been the first.”
“Wesley was really excited to be the first child in the country to take this medicine,” McDade says. To date, St. Jude has enrolled seven patients in the study.
One year into the trial, McDade is encouraged by the response of St. Jude patients. “So far, our patients have not encountered any significant side effects,” she says. “It appears to work in about 75 percent of children who take it. The preliminary results are promising, because it seems to be a safe and easy-to-tolerate medicine.”
Children take the medication by mouth once a day, instead of receiving a several-hour infusion every few weeks, which has been the typical treatment for chronic ITP in children.
McDade is quick to point out that the medication does not cure ITP, and it works only if the patient takes it regularly.
“This medication is just stimulating the body to make more platelets to compensate for the ones that are being destroyed,” she explains. “It’s a nice alternative, because the children can take a medicine once a day, achieve a stable platelet count and do the types of activities that might otherwise be limited.”
With his platelet count vastly improved, 15-year-old Wesley has returned to most of his regular activities. The only inconvenience is that he cannot eat dairy products within four hours of taking the medication. “I’m forgetful sometimes,” admits Wesley, who has the typical teenage addiction to cheese pizza and ice cream. But that’s a small price to pay for the ability to do normal activities, such as running road races and serving as the coach’s assistant for his high school’s football team. His family no longer worries incessantly about internal bleeding as a result of minor bumps or accidents.
“I want Wesley to feel as normal as possible and to experience the fun things that occur during high school,” his aunt says. “There’s always the chance that a rare accident could happen. But if it does, I know that this medication could make the difference in saving his life.”