Hydroxyurea (hydroxycarbamide) for transfusion-dependent β-thalassaemia

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#blood-transfusion #hematinics #humans #hydroxyurea #beta-thalassemia

Hydroxyurea (hydroxycarbamide) for transfusion-dependent β-thalassaemia

Ansari et al., 2019 | Cochrane Database Syst Rev | Systematic Review

Citation

Ansari Saqib H, Lassi Zohra S, ... Shamsi Tahir S. Hydroxyurea (hydroxycarbamide) for transfusion-dependent β-thalassaemia. Cochrane Database Syst Rev. 2019-Mar-16;3(3):CD012064. doi:10.1002/14651858.CD012064.pub2

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.

Key Findings

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 maint

Outcomes Measured

  • Requires manual extraction

Population

Field Value
Population See abstract
Sample Size See abstract
Age Range See abstract
Condition See abstract

MeSH Terms

  • Blood Transfusion
  • Hematinics
  • Humans
  • Hydroxyurea
  • beta-Thalassemia

Evidence Classification

  • Level: Systematic Review
  • Publication Types: Journal Article, Research Support, Non-U.S. Gov't, Systematic Review
  • Vertical: iron

Provenance

Source extracted via PubMed E-utilities API on 2026-04-09