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Sickle-cell disease treatment showing high success rate
August 7, 1996
Web posted at: 8:20 p.m. EDTFrom Correspondent Andrew Holtz
ATLANTA (CNN) -- Children affected by sickle-cell disease once suffered from the painful affliction without possibility of ever being cured.
But today there is hope, thanks to bone marrow transplants that have successfully eliminated the disease's complications for some patients.
Sickle-cell disease is a genetic disorder that strikes about one in 400 people of African descent. Tangela Roberts once had it.
"It was real bad for her at that time," said her mother, Angel Roberts. "I was scared. I didn't know what to do, and the doctor kept giving her morphine, codeine, and she was still having the pain crisis and everything."
But a bone marrow transplant five years ago changed Tangela from a sickly child, whose life revolved around pain and hospitals, to a normal little girl who now plays happily with her friends.
Sickle-cell disease is an illness in which sickle-shaped red blood cells clog vessels, causing pain, strokes and other damage.
A report in the New England Journal of Medicine offers the best look yet at using bone marrow transplants to cure sickle-cell disease. A group of researchers gave transplants to 22 children with the disease. Two of them died, but 16 appear to be cured.
"These are children that were doing very poorly, and they're basically cured of the disease," said Dr. James Eckman of Emory University.
When Tangela Roberts' parents decided to try a transplant five years ago, the benefits and risks of the operation were much more uncertain. Her parents worried about whether they were doing the right thing. (136K AIFF or WAV sound)
Doctors at the Medical University of South Carolina destroyed Tangela's diseased bone marrow with chemotherapy. Then they replaced it with marrow donated from her older brother, Paul. The healthy marrow produces normal blood cells, and her disease is gone.
"It has been a night-and-day difference in her quality of life. She has gone from a girl who was constantly crying, afraid, in the hospital at least once a month, to somebody who is living a normal life," said Dr. Migel Abboud, one of her doctors at the Medical University of South Carolina.
For doctors to suggest this major surgery, and for parents to agree to put their child through it, they must first evaluate the child's current quality of life. Is the patient likely to die young, to survive for decades -- or something in between?
They must also find a donor. Only about 5 percent of sickle-cell patients have a sibling or other relative who can donate healthy bone marrow that their bodies will accept.
Drugs and other treatments are also dramatically improving and lengthening the lives of people with sickle-cell disease. But a few, like Tangela, can leave it completely behind.
"She's healthy now," her mother said. "She's doing good. We haven't had any problems, you know -- we don't even go to the hospital."
To a child born sick, it is a sweet freedom.
Related sites:
- New England Journal of Medicine
- Medical University of South Carolina
- Guidelines: Sickle cell disease
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