Hydroxyurea for sickle cell disease
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Hydroxyurea is taken daily by mouth (orally).
How It Works
Hydroxyurea increases fetal hemoglobin (HbF) production and slightly raises the total hemoglobin concentration in the body. Fetal hemoglobin reduces the chance that red blood cells will sickle in a person who has sickle cell disease. So increased production of HbF can reduce the occurrence of sickling-related complications.
Why It Is Used
Hydroxyurea is used to reduce complications of sickle cell disease in adults who have a history of severe symptoms, including:
- Frequent painful events (at least three in a year).
- A history of recurrent acute chest syndrome.
- Severe anemia.
Taking hydroxyurea may prolong life for some people who have severe sickle cell disease.1
How Well It Works
Hydroxyurea can reduce the number of painful events and the recurrence of acute chest syndrome. It also can reduce hospital stays and the need for blood transfusion in adults who have sickle cell disease. And it can prolong the life of severely affected adults who respond to treatment. This medicine doesn't work for everyone though.
Hydroxyurea is still an experimental treatment for young children with sickle cell disease. So far, research shows that hydroxyurea is as effective for children as for adults. It may also reduce the risk of childhood strokes.2 More research is needed about long-term side effects.
Rare side effects with hydroxyurea include:
- Stomach problems, such as diarrhea and upset stomach.
- A rash or redness of the skin on the face.
- Bleeding problems.
- Reduced red blood cell production.
When hydroxyurea is used properly, these side effects are very uncommon.
Long-term effects (such as the risk of cancer) of this drug are unknown and are currently the subject of long-term research. The benefits of hydroxyurea may outweigh these long-term risks. Talk to your doctor about whether this medicine is right for you.
See Drug Reference for a full list of side effects. (Drug Reference is not available in all systems.)
What To Think About
Hydroxyurea doesn't cure sickle cell disease, but it can reduce the number of painful events and other complications and may prolong a person's life.
People who are taking hydroxyurea need frequent blood tests to monitor for adverse effects on bone marrow function.
If you have leg ulcers, talk to your doctor about whether this medicine is right for you.
Both men and women who are sexually active must take precautions to prevent pregnancy while taking hydroxyurea.
Hydroxyurea is still considered an experimental treatment in young children. Long-term effects of the drug on children are not yet known.
- Steinberg MH, et al. (2003). Effects of hydroxyurea on mortality and morbidity in adult sickle cell anemia. JAMA, 289(13): 1645–1651.
- DeBaun MR, Vichinsky E (2007). Hemoglobinopathies. In RM Kliegman et al., eds., Nelson Textbook of Pediatrics, 18th ed., pp. 2025–2038. Philadelphia: Saunders Elsevier.
Last Updated: December 9, 2008