Adult drug for sickle cell anemia may be safe for children
Research Activities, June 2009, No. 346
In 1998, the U.S. Food and Drug Administration approved the medication hydroxyurea for adults with sickle cell anemia. This inherited blood disease causes red blood cells to lose their shape, block circulation, and cause organ damage. Hydroxyurea is used to prevent sudden, painful episodes, called crises, caused by blocked blood flow to bones, lungs, abdomen, or joints. Although the drug has not been approved for pediatric use, in 2002 the National Heart, Lung, and Blood Institute recommended its use for children with sickle cell anemia.
Researchers from the Johns Hopkins Evidence-based Practice Center examined 26 published studies to determine the drug's effectiveness and safety for children. One randomized clinical trial of children with sickle cell anemia found that children who were given hydroxyurea had fewer and shorter hospitalizations compared with children who received a placebo. In three of four observational studies, the number of pain crises decreased when children took the drug.
Children who have sickle cell anemia tend to grow slower and reach puberty later than other children. A panel of experts found that hydroxyurea did not cause growth delays in children who were 5 to 15 years old. However, because the drug affects the reproductive system of male mice, it may have an adverse effect on sperm production after puberty. The authors suggest that frequent monitoring and coordination of care between primary care providers and pediatric hematologists may help improve the access of children to hydroxyurea's benefits. This study was funded by the Agency for Healthcare Research and Quality (Contract No. 290-02-0018).
See "Hydroxyurea for sickle cell disease: A systematic review for efficacy and toxicity in children," by John J. Strouse, M.D., Sophie Lanzkron, M.D., Mary Catherine Beach, M.D., M.P.H., and others in the December 2008 Pediatrics 122(6) pp. 1332-1342.