A new treatment for sickle-cell anemia--a drug that reduces the anemia and helps prevent the painful "crises" that mark the disease--has shown promise in a small, preliminary study of three patients with the disease.
In people with sickle-cell anemia, red blood cells contain an abnormal form of hemoglobin, the oxygen-carrying protein, that can distort cells from their normal bagel shape into a crescent or sickle shape, making them unusually fragile and often leading to painful blockages of blood vessels.
A report in last week's issue of the New England Journal of Medicine shows that the drug, hydroxyurea, can improve the survival of red blood cells and reduce pain in people with the disease. About 50,000 Americans have sickle-cell anemia.
The drug works by increasing the body's production of another form of hemoglobin that is normally produced during fetal life.
Hydroxyurea has been used for about 15 years to treat chronic leukemia and other blood disorders sometimes seen in elderly people. The report's authors said they were excited by the findings, but cautioned that before hydroxyurea becomes widely used for sickle-cell anemia, their results should be confirmed in a much larger, multihospital study that would compare the drug with a placebo.