Review Article

Annals of Hematology

, Volume 92, Issue 3, pp 289-299

Hydroxyurea treatment in β-thalassemia patients: to respond or not to respond?

  • Mehdi BananAffiliated withGenetics Research Center, University of Social Welfare and Rehabilitation Sciences Email author 

Rent the article at a discount

Rent now

* Final gross prices may vary according to local VAT.

Get Access


Hydroxyurea (HU) is a drug that induces fetal hemoglobin production. As a result, HU is widely used to treat β-thalassemia (β-thal) patients. However, the response of these patients to HU varies. Some β-thal patients respond favorably to treatment while others do not respond at all. HU has a number of side-effects and therefore its targeted prescription is beneficial. Hence, identifying the genetic determinants which lead to the differential HU response is important. This review summarizes recent findings which have shed light on this topic. Special emphasis is given to the mechanisms and genetic loci which may govern these differences. These findings have helped identify several single nucleotide polymorphisms which associate with the response to HU in both β-thal and sickle cell disease patients.


β-thalassemia Hydroxyurea Pharmacogenomics HbF