Skip to main content

Sickle Cell Drug Effective in Infants

A new study finds that a drug used to treat the pain of sickle cell disease in adults shows promise for use with children as well.

A drug used to prevent excruciating pain in adults with sickle cell disease appears to also be effective in infants and children suffering from the inherited blood disorder. Dr. Russell Ware, professor of pediatrics in the division of hematology-oncology at Duke University Medical Center, was lead investigator on the study.

"We treated a number of children, some of them very young infants -- as young as six months old."

Researchers gave the children a liquid form of the drug hydroxyurea over a two-year period. Ware says the results were very promising:

"They tolerated hydroxyurea very well. The side effects were minimal and very well-tolerated and there were some impressive hematological responses."

Ware says the hydroxyurea also appears to delay or perhaps even prevent the organ damage usually associated with sickle cell disease. He cautions that this was a very preliminary study, and it will be some time before physicians can consider using hydroxyurea to treat children with sickle cell disease.

I'm Tom Britt for MedMinute.