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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
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Fuente et al. (2001) studied this question.
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