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| |    Hydroxyurea Treatment for Sickle Cell Disease by Lewis Hsu, M.D., Ph.D. reviewed by Laura Jana, M.D., F.A.A.P. As you may already know, hydroxyurea is the first effective medication (out of hundreds that have been tried) for treatment of sickle cell disease. While early tests of the medication mainly focused on its benefits for adults, recent pediatric studies--some published, some not--show that hydroxyurea also lessens the complications of sickle cell disease in children. Worries that hydroxyurea might interfere with growth or development has not been borne out in study findings, leading to a wider acceptance of its use in pediatric patients with sickle cell. Many centers treat school-age children and adolescents; some are studying the effects of hydroxyurea on infants and preschoolers. No long-term side effects have been reported because of the general newness of the medication. Presently, only a handful of patients have been treated for more than 12 years; most studies involve people with sickle cell who have been on hydroxyurea for less than 6 years. What is hydroxyurea? Hydroxyurea is a chemical compound that has been used as a medication for more than a century. It was one of the first medicines used in the fight against cancer, and it's still a valuable drug for some of the slower-growing types of the disease. For sickle cell, it comes in capsule form and is administered once a day. Hydroxyurea is not a cure, but it can make sickle cell disease milder. In many people, it helps cut the number of pain episodes, acute chest problems, and hospitalizations in half. New findings also show that it improves the life expectancy of adults with sickle cell. Although researchers aren't sure why it works, one of hydroxyurea's effects is to make the body produce red blood cells with more fetal hemoglobin, which dilutes the sickle hemoglobin inside the red blood cells. It also seems to produce cells that are larger, less dehydrated, more flexible, and less sticky-all highly desirable properties. The number of white blood cells also goes down on hydroxyurea, which seems to be a good thing for people with sickle cell. When patients stop taking hydroxyurea, they generally revert to their prior health status. This demonstrates that there is no permanent change in the body, and the drug must be given long term (at least until a better treatment is found). The flip side to benefits As with most medications, hydroxyurea presents potential risks as well as benefits. Possible long-term side effects are the increased risks of both cancer and birth defects. A link has been found between hydroxyurea treatment and a slightly higher rate of leukemia for people without sickle cell disease who have blood disorders that predispose them to leukemia. The worries about birth defects are based on animal studies. However, neither a higher cancer rate nor an increase in birth defects has been reported in people with sickle cell disease who are receiving hydroxyurea. Currently, several hundred adults with sickle cell in the United States are on the medication, and a registration system is in the works to track long-term side effects. Practical considerations Hydroxyurea treatment generally is reserved for children whose sickle cell disease is particularly severe, requiring frequent hospitalizations for pain or lung problems. In the beginning of the treatment phase, blood counts typically are checked every two to four weeks, as a lower-than-usual count is a potentially problematic side effect. Slow, careful adjustments to the child's dosage take place over the span of a few months to ensure the best treatment possible. Most hydroxyurea patients find it extremely helpful in easing the severity of their sickle cell disease. Some children who find treatment worthwhile had been subject to monthly hospitalizations before being given hydroxyurea; now they face only about two such stays a year. Some teenagers, however, eventually quit the treatment after just a few months because they consider the daily obligation and frequent lab tests too inconvenient.
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