Examples

Generic Name	Brand Name
hydroxyurea	Droxia, Hydrea

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 with sickle cell disease. Increased production of HbF can therefore 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 also prolong life for some people who have severe sickle cell disease.¹

How Well It Works

Hydroxyurea reduces the number of painful events and the recurrence of acute chest syndrome. It also reduces hospital stays and the need for blood transfusion in adults with sickle cell disease. And it can prolong the life of severely affected adults who respond to treatment.

Between 10% and 25% of adults treated with hydroxyurea don't respond to treatment.²

Hydroxyurea is still an experimental treatment for young children with sickle cell disease. Preliminary research shows that hydroxyurea is as effective for children as for adults. It may also reduce the risk of childhood strokes. Results aren't available on the long-term side effects of hydroxyurea on children.^{3, 4}

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.

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Citations

1. Steinberg MH, et al. (2003). Effects of hydroxyurea on mortality and morbidity in adult sickle cell anemia. JAMA, 289(13): 1645–1651.

2. Steinberg MH (1999). Management of sickle cell disease. New England Journal of Medicine, 340(13): 1021–1030.

3. Wang WC, et al. (2001). A two-year pilot trial of hydroxyurea in very young children with sickle cell anemia. Journal of Pediatrics, 139(6): 790–796.

4. Wang WC, et al. (2002). Effect of hydroxyurea on growth in children with sickle cell anemia: Results of the HUG-KIDS Study. Journal of Pediatrics, 140(2): 225–229.