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. 2019 Mar 16;3(3):CD012064.
doi: 10.1002/14651858.CD012064.pub2.

Hydroxyurea (hydroxycarbamide) for transfusion-dependent β-thalassaemia

Saqib H Ansari  1 , Zohra S Lassi, Salima M Khowaja, Syed Omair Adil, Tahir S Shamsi
Affiliations

Hydroxyurea (hydroxycarbamide) for transfusion-dependent β-thalassaemia

Saqib H Ansari et al. Cochrane Database Syst Rev. .

Abstract

Background: Hydroxyurea (hydroxycarbamide) promotes the production of foetal haemoglobin (HbF) by reactivating gamma-genes. Evidence has shown clinical benefits of hydroxyurea in people with sickle cell anemia; however, only a few studies have assessed this treatment in people with beta (β)-thalassaemia.

Objectives: The primary objective is to review the efficacy of hydroxyurea in reducing or ameliorating the requirement of blood transfusions in people with transfusion-dependent β-thalassaemia. The second objective is to review the safety of hydroxyurea with regards to severe adverse effects in this population.

Search methods: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Haemoglobinopathies Trials Register, compiled from electronic database searches and hand searching of journals and conference abstract books. We also searched electronic databases and trial registries, including ClinicalTrials.gov, the WHO ICTRP and PubMed (09 October 2018).Date of last search of the Group's haemoglobinopathies trials register: 04 March 2019.

Selection criteria: Randomised controlled trials of hydroxyurea in people with transfusion-dependent β-thalassaemia, compared with placebo or standard treatment or comparing different doses of hydroxyurea.

Data collection and analysis: Two authors independently assessed trials for inclusion in the review, which was verified by a third author.

Main results: No trials were eligible for inclusion in this review.

Authors' conclusions: Currently, there is no high-quality evidence to support or challenge the continued use of hydroxyurea for managing people with transfusion-dependent β-thalassaemia. Multicentre, randomised controlled trials (compared to placebo or other available treatment, i.e. blood transfusion and iron chelation) are needed in order to assess the efficacy and safety of hydroxyurea for reducing the need for blood transfusion, for maintaining or improving mean haemoglobin levels, as well as for determining its cost-effectiveness.

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Conflict of interest statement

Saqib Ansari: none known.

Zohra Lassi: none known.

Salima Muhammad Khowaja: none known.

Syed Omair Adil: none known.

Tahir Shamsi: none known.

Figures

1
1
Study flow diagram.
See this image and copyright information in PMC

Update of

  • doi: 10.1002/14651858.CD012064

References

References to studies excluded from this review

Alebouyeh 2004 {published data only}
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Bordbar 2014 {published data only}
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References to ongoing studies

NCT03183375 {published data only}
    1. NCT03183375. The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan. www.clinicaltrials.gov/show/NCT03183375 Date first received: 05 June 2017.
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