Hydroxyurea           Chronic Myeloid Leukemia           CML


  • Hydroxyurea (Hydrea® or Litalir® in some countries, Bristol-Myers Squibb Oncology) was introduced for the treatment of CML in 1972.
  • Hydroxyurea is indicated for the treatment of CML and several other malignancies.
  • Hydroxyurea therapy is superior to busulfan in CML:
  • A large randomised study of 458 patients with CML in chronic phase found that patients treated with hydroxyurea had a significantly longer median survival.
  • This survival advantage was present in all prognostic sub-groups.
  • Hydroxyurea has a better toxicity profile than busulfan.
  • It provides rapid but more transient control of haematological manifestations and thus requires frequent follow-up.
  • Its main side-effects include:
    • Nausea
    • Vomiting
    • Diarrh<ea
    • Mucosal ulcers
    • Skin reactions, including ulceration
Hydroxyurea is a mainstay of current CML treatment. It rapidly lowers WBC counts and can be given orally. Its use does not appear to affect the results of subsequent treatment with SCT or IFN-α. It is given in doses of 40 mg/kg daily and adjusted to maintain leukocyte counts in the range of 2–10 x 109/L.

Extract of CML-INFO.com