Children with Graves Disease At-Risk from Propylthiouracil

Newswise — Of the 4000 children with Graves' disease treated in the U.S. annually, 40 percent are routinely treated with the drug propylthiouracil. Unfortunately, some of these children will suffer liver failure induced by this drug.

Writing in the current issue of the New England Journal of Medicine, Scott A. Rivkees, MD, of the Yale University School of Medicine and Donald R. Mattison, MD, of the Eunice Kennedy Shriver National Institute of Child Health and Human Development, call for an end to its use.

"We suggest that propylthiouracil should not longer be used as first-line treatment for Graves' disease in children," says Dr. Rivkees. "More children receive methimazole than propylthiouracil and there are no reports of liver failure or liver transplantation in association with methimazole."

According to Dr. Mattison, there may be 1000 children in the U.S. currently receiving propylthiouracil and there is no good plan for managing their risk of hepatoxicity other than not using the drug. At least one of these 1000 children will develop propylthiouracil-induced liver failure and require liver transplantation, he says.

In 2007, more than 9000 prescriptions were written of the pediatric use of propylthiouracil in the U.S. Based on data from the Food and Drug Administration and data presented at a 2008 workshop discussing the safety of the use of the drug in children, routine liver function surveillance has not been effective in measuring the risk.

"The number of pediatric patients who have suffered reversible liver injury may be as high as ten times the number of those with irreversible damage," explains Dr. Rivkees. "Alternative treatment should be considered."

They concluded that the apparent good safety record of methimazole suggests that it, and not propylthiouracil, should be the first line of therapy for children.