Medical Advancement
Marrow transplant cures adult sickle cell disease
An electron microscopic view of fragile red blood corpuscles seen in sickle cell disease. Photo: REUTERS
Bone marrow transplants, already used to treat some children with sickle cell disease, also may cure some adults with this deadly genetic defect that causes red blood cells to contort, U.S. scientists said recently. Nine of 10 adult patients given an experimental bone marrow transplant treatment were cured of sickle cell disease, researchers at the U.S. government's National Institutes of Health reported in the New England Journal of Medicine. If the early results hold, the treatment "could be ideal for patients with severe sickle cell disease," Dr. Miguel Abboud of American University of Beirut Medical Center in Lebanon said in an editorial accompanying the study. Such transplants already are used to cure children with the disease who have a compatible donor who provides bone marrow. Bone marrow gives rise to blood cells. Destroying a patient's bone marrow and replacing it with healthy marrow from a donor, often a sibling, is considered too risky for adults. In conventional bone marrow transplants, doctors try to destroy all of a patient's own bone marrow. Using the new technique, adults are given a lower dose of radiation, only partially destroying the patient's bone marrow. This approach leaves enough space inside the patient's bones for the donated marrow to find a home and produce enough healthy red blood cells to compensate for the defective ones. In sickle cell disease, an inherited disorder, blood cells become stiff and sickle-shaped, causing them to block blood vessels and starve tissues of oxygen. It had been thought that by the time people with sickle cell disease become adults, they had have suffered too much kidney, lung and liver damage to allow for a safe transplant.
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