A major international study has shown that bone marrow transplants can cure sickle cell disease in some children, a finding that experts say could herald a new era in treating the blood disease.
Researchers involved in the study said they had achieved the cure in 16 of 22 patients with advanced disease by replacing the bone marrow that produces the defective red blood cells characteristic of the disease. Two of the children died after undergoing transplants.
Sickle cell disease is a family of inherited and previously incurable disorders that afflict several million people around the world. The disease is most common among people whose ancestors came from Africa, the Middle East, the Mediterranean or India.
In the United States, the condition primarily afflicts an estimated 80,000 African-Americans, and 1 in 12 black babies is born with a genetic tendency for passing on the disease.
In its worst form, the disease can result in severe episodes of pain in various parts of the body, stroke, damage to internal organs and death. Present treatments, which can only relieve symptoms, include blood transfusions, painkillers and other drugs.
Although the study involved only children under age 14 who received donated marrow from siblings with highly compatible tissue, researchers said they hoped for a cautious expansion to a wider range of patients.
Experts said the bone marrow transplants are risky procedures that carry a 10 percent risk of death under the best of circumstances. This risk -- along with complications from the operation, including infertility resulting from drugs used to destroy the old, defective bone marrow -- means that the treatment cannot be considered for all sickle cell patients, the experts said.
The study, being published today in the New England Journal of Medicine, is significant because it proves, in a well-designed trial, that marrow transplants can cure sickle cell disease, as other smaller studies had indicated. And finding a way to cure even a small percentage of sickle cell cases is a major development, specialists said.
"Our findings indicate that bone marrow transplantation can cure sickle cell disease in a significant number of patients with advanced disease," said Dr. Keith Sullivan of the Fred Hutchinson Cancer Research Center in Seattle, the study's lead author. "In the paper, we used the word 'curative' with a great degree of consideration and careful look at the data."
