Sickle cell anemia drug might protect children
Posted May 13, 2011 1:54 p.m. EDT
Durham, N.C. — A drug approved for adults might be able to help the one in 500 African-American children who are born with sickle cell anemia, leading to a shorter life-span and more days spent in pain and in hospitals.
Six-year-old Caleb Davis is one of the children has benefitted from the drug.
"He runs. He plays. He's involved in sports. He does karate. He's not limited at all," his mother, Casonja Davis, said.
But Caleb has sickle cell anemia, which began to cause problems for him a little more than a year ago.
"He had his first pain crises and was hospitalized for pneumonia," Davis said.
In the disease, abnormal hemoglobin turns the red blood cells into crescent, or sickle, shapes, which are sticky and stiff and block blood flow. That can cause pain in the limbs, serious infections and organ damage.
"The first thing we do is give them penicillin to prevent infections," said Dr. Courtney Thornburg, with the Duke University Department of Pediatrics.
For the past year, Caleb has also been on hydroxyurea, a drug shown to be effective at preventing complications of sickle cell in adults.
Thornburg was a lead researcher in a national study called Baby Hug that looked at the effectiveness of hydroxyurea in children starting at age 1 before sickle cell anemia starts to damage the body.
The drug could let us "be more proactive, rather than reactive," Thornburg said.
She said the drug was well tolerated in the young study participants.
"We see the changes from these sickle cells to nice round healthy cells," Thornburg said.
Since Caleb's been on the drug, his mom has noticed improvements.
"After the hydroxyurea this year, in all he's maybe missed five to 10 days of school as opposed to in kindergarten, he missed about half of his school year," Davis said.
Thornburg said she hopes the results of the study will cause more doctors to consider prescribing the drug for their young patients identified with the disease.
The study is published in the May 14 issue of the journal Lancet.