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

Genetics Research Center, University of Social Welfare and Rehabilitation Sciences, Evin, Daneshjoo Blvd., Koodakyar St, Tehran, Iran, .
Annals of Hematology (Impact Factor: 2.63). 01/2013; 92(3). DOI: 10.1007/s00277-012-1671-3
Source: PubMed


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.

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Available from: Mehdi Banan, Oct 10, 2015
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    ABSTRACT: Background: β-Thalassemia is an inherited hemoglobin disorder caused by defective synthesis of ß-globin chains. Hemoglobin (Hb) F induction is a possible therapeutic approach which can partially compensate for α and non-α globin chains imbalance. Objectives: We aimed to investigate the efficacy and safety of Hydroxyurea (HU) in diminishing transfusion requirements of patients with β-thalassemia major in Southern Iran. Patients and Methods: In this single-arm clinical trial, all transfusion-dependent β-thalassemia patients older than two years old (n = 97) who had inclusion criteria of the study and had been registered for at least six months in Dastgheib thalassemia outpatient clinic (a referral center affiliated to Shiraz University of Medical Sciences) were evaluated from October 2010 to December 2011. The patients were treated with HU with a mean dose of 10.5 mg/kg for a mean duration of 8 months (range 3-14 months). Transfusion needs and Hb levels were compared before and after HU treatment. Results: The mean volume of blood transfusion decreased significantly following HU treatment (0.71 mL/kg/day vs. 0.43 mL/kg/day, P < 0.001). Two-thirds of the patients showed good and partial response. No serious adverse reaction was observed except persistent neutropenia in two patients. Conclusions: Hydroxyurea can be safely used in some transfusion-dependent β-thalassemia patients to decrease their transfusion needs.
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