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Is hydroxycarbamide effective in sickle cell disease?

Hydroxycarbamide is an oncolytic drug which is used to treat sickle cell disease. Research has shown that hydroxycarbamide reduces the frequency of the pain episodes inherent in severe forms of this disease by an average of 45%. Hydroxycarbamide also reduces the incidence of acute chest syndrome, and the need for blood transfusions. An association between the use of hydroxycarbamide and enhanced survival rates has only been shown in cohort studies. These results only concern patients with the usual forms of sickle cell disease. The efficacy for certain subforms of sickle cell disease, such as HbSC, has not been proven. No studies have as yet shown whether hydroxycarbamide has any adverse effects in the long term which limit its use. Frequent haematological monitoring is necessary to determine the correct dosage of hydroxycarbamide.

  • Research findings show that there is good evidence for prescribing hydroxycarbamide to patients with severe sickle cell disease.
  • An association has been found between the use of hydroxycarbamide and prolonged survival of patients with severe sickle cell disease, but conclusive evidence is as yet lacking.
  • There is insufficient information available about its teratogenicity and possible long-term adverse effects.
  • When prescribing this drug, doctors must, in consultation with the patient or their parents, carefully weigh up the potential advantages and disadvantages of the use of hydroxycarbamide.

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  • Hajo I. Wildschut, dr
  • Sanne van der Heijden, MSc, pharmacist Stichting Geneesmiddelenbulletin, Utrecht