Hydroxyurea was developed in the 1960s as a possible anti-cancer agent. It won approval for treatment of adults and later adolescents with sickle cell anemia in 1998 following evidence that the drug reduced episodes of severe pain and improved patient quality of life.
The drug works by increasing production of fetal hemoglobin, a form of the oxygen-carrying protein that is unaffected by the mutations that cause sickle cell disease. Fetal hemoglobin normally drops dramatically after birth. Hydroxyurea, however, increases production of red blood cells that contain that form of hemoglobin.
The drug remains an underutilized treatment for sickle cell anemia. Wang estimated the drug is prescribed to about 30 percent of pediatric patients nationwide and an even smaller percentage of adults. Work is underway at St. Jude and other medical centers to identify and address barriers to more widespread use of the drug, including lingering concerns about possible long-term toxicity.
The senior author is Scott Grosse of the U.S. Centers for Disease Control and Prevention (CDC). The other authors are Suzette Oyeku, Montefiore Medical Center, Bronx, N.Y.; Zhaoyu Lou, Billie Fish and Bruce Thompson, all of Clinical Trials and Surveys Corp., Owings Mills, Md.; Sheree Boulet, CDC; Scott Miller, SUNY-Downstate Medical Center/King's County Hospital Center, Brooklyn; and James Casella, Johns Hopkins University School of Medicine, Baltimore.The research was funded in part by the National Heart, Lung, and Blood Institute and the National Institute of Child Health and Human Development. SOURCE St. Jude Children's Research Hospital