• E-mail
• Print
• Comment
• Font Size
• Digg
• del.icio.us
• Discuss article

Sickle Cell Study Halted

Posted on: Thursday, 29 December 2005, 09:00 CST

By Rebecca Vesely, STAFF WRITER

Some children with sickle cell disease need blood transfusions to prevent strokes and should not halt the treatment despite side effects, according to a study released today that included Children's Hospital and Research Center Oakland.

The study, published in the New England Journal of Medicine, dashed hopes that some children with sickle cell disease could discontinue blood transfusions under careful monitoring. The study was cut short when two children enrolled in the trial developed strokes after stopping monthly blood transfusions.

Regular transfusions have long been standard treatment for children with sickle cell anemia at risk for stroke. About one in 10 patients under age 20 with sickle cell anemia get strokes, though scientists are

not exactly sure why. The transfusions can cut the risk of stroke by 90 percent.

In the late 1990s, researchers led by Dr. Robert Adams of the Medical College of Georgia found a sonogram test that detects the velocity of blood vessels could accurately estimate which children with sickle cell anemia were at risk of stroke.

Parents of children found to be at risk for strokes were advised to start monthly blood transfusions -- the only known preventative treatment.

In the new study, Adams and other researchers wanted to see whether children undergoing regular transfusions could stop the treatment -- provided they were monitored for stroke risk using the sonogram test.

Dr. Keith Quirolo, a sickle cell expert at Children's Hospital Oakland, where children were enrolled in the trial, said researchers wondered if children would pass a point where they needed the procedure.

"We wanted to know, was there a critical time when you transfused kids, then stopped the transfusions and they wouldn't be at risk for stroke," Quirolo said.

A dangerous side effect of transfusions is the build-up of iron, which, over time, can damage tissues and organs and lead to death. To prevent the buildup of iron, patients receiving regular transfusions require an overnight infusion under the skin five to six nights a week.

The trial was comprised of 79 children who had been receiving transfusions for about three years. Of those enrolled,

41 randomized children stopped transfusions, while 38 continued to receive them.

In the group that stopped getting transfusions, 14 became at high risk for developing stroke and two children did have strokes.

"The study showed that it wasn't safe to stop the transfusion after three years," Quirolo said.

For children who are at risk for stroke and have sickle cell disease, transfusions can lead to a normal life.

Nikita Williams, 15, of Oakland has undergone monthly blood transfusions at Children's Hospital Oakland since age 11, when she suffered a mild stroke and was in intensive care with other complications for two months.

Her mother, Erica Cloud, said in a phone interview that the transfusions make her daughter feel energetic.

"The blood transfusions keep her out of crisis," Cloud said. "I say thanks to the blood transfusions. She wasn't doing well without them."

Williams is now taking a new, less invasive drug called Exjade to remove excess iron from her system. Manufactured by Novartis, Exjade was approved by the Food and Drug Administration in November and is taken orally once a day dissolved in water or orange juice.

Sickle cell disease is a genetic disorder where red blood cells form into crescent shapes, causing them to block blood vessels. The disease can lead to numerous health complications and severe pain. About 1 in 650 African Americans and about 1 in 1,000 Latinos nationwide have sickle cell disease.

Researchers are now looking at genetic factors that predispose children with sickle cell anemia to strokes, Quirolo said.

"The way they're going to cure stroke is to find out genetically what causes it," he said.

Source: Oakland Tribune

More News in this Category