Hydroxyurea is a medicine that can help people who have sickle cell disease. It keeps blood cells round and flexible, so they flow better and deliver oxygen to the body more easily. This means less pain and fewer trips to the hospital.
Sickle cell disease makes red blood cells become C-shaped instead of round. These sickle-shaped cells get stuck and block the blood flowing inside small blood vessels. This can be painful. It can lead to other problems that need to be treated in the hospital.
Sickle cells also break down more quickly than healthy red blood cells. Having too few red blood cells can lead to anemia. People with anemia feel tired and have less energy.
Hydroxyurea (hy-drok-see-yer-EE-uh) helps the body make a type of hemoglobin that helps keep red blood cells round. Hemoglobin is the protein inside red blood cells that carries oxygen to the body. Round and flexible red blood cells work better and last longer. So people who take hydroxyurea can have more energy, and less pain and anemia. They often spend less time in the hospital.
Hydroxyurea can help people with sickle cell disease, including those who have:
Children as young as 9 months old can take hydroxyurea.
Doctors sometimes use hydroxyurea to treat other kinds of health problems, not just sickle cell disease. Like many medicines, it has different uses. In high doses, it can help with some kinds of blood cancers. The dose used to treat sickle cell disease is very low.
Hydroxyurea comes as either a liquid medicine or a small capsule that your child or teen swallows one time each day. It works best when they take it at the same time every day. Some people find it helps to set an alarm to stay on schedule.
Before your child starts taking the medicine, your care team will order a blood test to figure out the starting dose. Then they’ll order monthly blood tests to see how the medicine is working. As hydroxyurea boosts a person’s healthy red blood cells, it sometimes can affect other blood cells. Regular blood tests help the care team make sure the dose is right. They can change the dose or stop the medicine, if needed.
It can take a few months to find the right dose. After that, your child will get blood tests less often.
Most people who take hydroxyurea do well and don’t notice any side effects. Those who do might have dark fingernails or headaches. Often, side effects go away quickly as a person adjusts to the new medicine.
Hydroxyurea is usually paid for by insurance. Your cost (if any) will depend on the type of insurance you have. Your child's doctor’s office will help you find out if there are costs for you.
To take a balanced look at costs, it helps to compare the planned costs of taking hydroxyurea with the unplanned costs of not taking it. Pain crises and anemia can lead to ER visits, hospital stays, and missed days of work or school. In addition to a person not feeling well, these problems can be expensive.
When deciding what’s best for your child, ask yourself questions like:
If the care team recommends hydroxyurea for your child, talk to them if you have questions or concerns. If your child is old enough, include them in the discussion too. Together you can make a choice you both can feel good about.
Reviewed by: Corinna L. Schultz, MD
Date Reviewed: Nov 1, 2022
