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Richard T. Silver MD Myeloproliferative Neoplasms Center

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Hydroxyurea

Hydroxyurea is an oral chemotherapy medication that is used as a “cytoreductive therapy” in myeloproliferative neoplasms (MPNs) to control the number of blood white cells, red cells, platelets, and to reduce spleen size, and phlebotomy requirements. Hydroxyurea works by inhibiting DNA synthesis and causing cell death. Hydroxyurea can cause white and red blood cells and platelets to decrease below normal values and this can increase the risk of infection, anemia, and/or bleeding. Hydroxyurea also goes by brand names Hydrea® and Droxia®.

In clinical trials, hydroxyurea has been shown to reduce the risk of abnormal blood clots in patients with essential thrombocythemia (ET) and polycythemia vera (PV). Hydroxyurea is often used in older, higher-risk patients in order to decrease the risk of thrombosis (blood clots). During treatment with hydroxyurea, routine blood work will be done to monitor side effects and response to therapy. You should report to your medical team any side effect you think you may be experiencing.

    Side Effects

    Common side effects:

    • Nausea
    • Vomiting
    • Loss of appetite
    • Mouth sores
    • Diarrhea
    • Constipation
    • Skin ulcers
    • Hair loss
    • Low blood counts

    Potentially Serious Side Effects:

    • Secondary Malignancies: In patients who receive long-term hydroxyurea, secondary leukemia or skin cancer can occur. There is a debate among clinicians about how much, if any, hydroxyurea increases the risk of leukemic transformation for patients with MPNs.
    • Pregnancy and fertility: Hydroxyurea should not be used during pregnancy and it can also affect fertility.
    • Blood Count Suppression: Hydroxyurea suppresses blood cell counts. If the blood counts become too low there is risk of infection or bleeding. This is usually reversible if the medication dose is reduced or stopped to allow your infection fighting cells and platelets to recover.

    Additional Information: https://medlineplus.gov/druginfo/meds/a682004.html

    Richard T. Silver MD Myeloproliferative Neoplasms Center 525 East 70th St., Starr Pavillion, 3rd Floor New York, NY 10021 SilverMPNCenter@med.cornell.edu