SALT LAKE CITY — A drug used to stop ongoing bleeding in severe trauma patients of all ages since the 1960s has never been clinically tested in children, according to doctors at Primary Children’s Hospital.
“It is one thing that we see often with medications in the U.S. and across the world, is pediatrics has long been neglected just for various reasons. We have seen that when drug manufacturers look at drugs, children just, they aren’t included in those studies,” said Nicholas Weaver, doctor of pharmacy at Primary Children’s Hospital.
That’s why the hospital is teaming up with three others across the country to test the drug tranexamic acid, or TXA, in children in the first study of its kind, funded by the National Institutes of Health.
Though doctors say the drug is safe for children, its effectiveness for them has evaded extensive clinical research until now.
The goal is to see how well it helps prevent bleeding in children; if it can help prevent the need for blood transfusion; if it can reduce deaths in injured pediatric patients; and to find the correct dosage for children.
If a child with a severe head or torso injury comes in with signs of ongoing bleeding, they will be randomly chosen to receive either one or two doses of TXA, or a saline placebo. Each child will then be followed long term by researchers to see what their neurologic outcomes are, if they needed a blood transfusion, how long they spent in the hospital and other outcome measures.
The drug must be administered within three hours of the injury, said Dr. Hilary Hewes, the site’s primary investigator for the University of Utah Health and Primary Children’s Hospital.
Because of the short time frame in which the child needs to receive the drug, the Food and Drug Administration has given the hospital permission to conduct emergency research. That means when a child comes to the hospital without a parent or guardian, the hospital can immediately enroll them in the study without a parent there. When the child’s parent or guardian arrives, they can remove the child from the study if they wish, Hewes said.
Parents can opt their children out in advance if they do not want them in the study should an emergency occur. For more information about the trial and opting out, visit tictoctrial.org.
Weaver said the drug works by preventing blood clots from breaking down. When blood clots do break down, hemorrhaging continues. He emphasized it’s meant to be used for major trauma to the head or torso caused by incidents like car crashes or major falls — not small cuts.
In adults, TXA has been found to reduce death and the need for blood transfusion.
However, Hewes said, “we don’t know the right dose for a child. We know there’s a big safety range, so we’re hoping to better understand the most effective dose to prevent bleeding, as well.”
Weaver said though the drug has been used on children for decades, it hasn’t been clinically tested because even as legislation has required the testing of newer drugs on children, the older TXA was never “forced through” clinical trials.
Quality research in the form of a double-blind placebo control trial — like the current study — is also difficult to receive approval for in children, Hewes explained, and requires a lot of money and time.
The pilot study will enroll 40 children across the four hospitals, which also include UC Davis Children’s Hospital in Sacramento, California, Nationwide Children’s Hospital in Columbus, Ohio, and Children’s Hospital of Philadelphia.
Hewes said that should the drug prove effective in children, it could potentially help between 10 and 30 kids a year at Primary Children’s Hospital alone.