Is hydroxyurea leukemogenic in children with sickle cell disease?

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Is hydroxyurea leukemogenic in children with sickle cell disease?

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Hydroxyurea (HU) has been used for more than 10 years in children severely affected with sickle cell disease (SCD) to reduce the number of painful episodes. Short- to medium-term tolerance of the drug is good.1-3 Concern remains, however, about the long-term safety of the drug and, in particular, its leukemogenic potential. Leukemia has been reported in myelodysplastic syndromes after at least 3 to 4 years of exposure to the drug,4-5 so only long-term studies including significant numbers of patients can resolve concern for SCD. So far, 3 studies on long-term effects of HU in SCD children have been published (Table 1).6-8 View this table: [in this window] [in a new window] Table 1. Long-term studies on hydroxyurea use in children with sickle cell disease Ferster et al8 refer to a malignancy observed in one SCD patient on HU, the details of which were published in an abstract reporting the European experience with HU use in SCD children.9 We believe it is important to emphasize that thi

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