What is the role of hydroxyurea in the treatment of chronic myelogenous leukemia (CML)?

Updated: May 23, 2021
  • Author: Emmanuel C Besa, MD; Chief Editor: Sara J Grethlein, MD, FACP  more...
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Hydroxyurea (Hydrea), an inhibitor of deoxynucleotide synthesis, is the most common myelosuppressive agent used to achieve hematologic remission. The initial blood cell count is monitored every 2-4 weeks, and the dose is adjusted depending on the WBC and platelet counts. Most patients achieve hematologic remission within 1-2 months.

This medication causes only a short duration of myelosuppression; thus, even if the counts go lower than intended, stopping treatment or decreasing the dose usually controls the blood counts. Maintenance with hydroxyurea rarely results in cytogenetic or molecular remissions.

European Society for Medical Oncology (ESMO) guidelines suggest that hydroxyurea (40 mg/kg daily) may be used as initial therapy, before confirmation of the BCR–ABL1 fusion in patients with immediate need for therapy because of high leukocyte counts or clinical symptoms. TKI therapy should be started immediately after confirmation of BCR–ABL1 positivity, and the hydroxyurea dose tapered before discontinuation. [22]

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