Monroe Carell Jr. Children's Hospital at Vanderbilt
Monroe Carell Jr. Children's Hospital at Vanderbilt
Monroe Carell Jr. Children's Hospital at Vanderbilt
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Monroe Carell Jr.
Children's Hospital
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Nashville, TN 37232

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Hydroxyurea Treatment


Hydroxyurea is a medication that can dramatically improve the quality of life for many sickle cell patients. It does not cure the disease, but it can improve many symptoms, by:

  • preventing pain episodes
  • preventing acute chest syndromes
  • decreasing the need for blood transfusions
  • decreasing the number of hospitalizations
  • improving survival

Hydroxyurea is not a drug that works immediately after one or two doses. It must be taken every day, along with folic acid, because the body makes new sickle cells every day. The effect of hydroxyurea will be felt after one or two months. Patients will begin to feel more energetic. The maximum benefit of hydroxyurea will be reached after about six months.

Is hydroxyurea safe?

Hydroxyurea is the only FDA-approved therapy to decrease the rate of pain, life-threatening lung disease, and hospital stays for those living with SCD. It has been successfully used to treat adult patients since 1998. Experience shows it is effective for children, but the dose for each patient must be carefully monitored with frequent blood tests. Too small a dose will not have the desired effect. Too large a dose will cause all blood cell counts to drop, even good cells.

Hydroxyurea is usually prescribed for patients aged 5 and older, with frequent pain episodes, very low red blood cell count, acute chest syndrome, or other sickle cell problems. It is not known yet whether hydroxyurea can prevent strokes.

How hydroxyurea works

Hemoglobin F (fetal hemoglobin) protects against sickle cell symptoms. It prevents hemoglobin S from sticking together and decreases the sickling of red blood cells. Patients with high hemoglobin F levels have fewer symptoms than those with low hemoglobin F levels. Hydroxyurea increases hemoglobin F and keeps the red blood cells round.

How Hydroxyurea works


Last Edited: December 6, 2016
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